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Speaking at a Fundraiser 29
Speaking at a Support Group 589
Speaking to a Congressman 49
Interview 20
Writing in a Newspaper 20
Writing in a Blog 32
Writing in a Website 103
Writing to a Congressman 45

This table shows the types of outreach efforts being undertaken by our NEALS Research Ambassadors. Through these, they are educating and advocating about ALS research to patients, clinicians, press and policymakers. We are very proud of them! If you would like to be a NEALS Research Ambassador please apply for the 2013 ALS Clinical Research Learning Institite here.
 
 

Clinical Research Learning Institute

 
HR 3116 MODDERN Cures Act
Posted by: Steve Stefano November 18, 2014
I would like to thank Perri and NEALS for the wonderful experience in Florida. In august 2013 Congressman Lance from New Jersey authored a bill in the US House of Representatives called HR 3116, The MODDERN Cures Act. This bill gives drug companies ten years exclusivity rights instead if seven. This bill will help to find cures for not only ALS but for incurable diseases. Since April 2014 I have been contacting US representatives in regards to HR3116. As of the beginning of November 2014 seven out of the eight Minnesota representatives have signed onto the bill. I am advocating passionately for this bill to pass. I have been working very closely with MollyMcDonnell, Congressman Lance’s administrative health assistant. She emailed me last Friday informing me that 78 out of the 435 members of the US House of Representatives have given their signature and support. She also gave me permission to give her email so people reading the blog who have questions about the bill can contact her. I am asking everyone in the ALS community to contact your members of congress within your states. We really need to get this bill passed! With the success of the ice bucket challenge, the awareness, and money raised this bill will be a game changer if passed into a law.
 
Thank you for your help and lets gets this done!
 
I and Molly encourage you to contact her for particulars of the bill and what it will take to get it passed into a law. Here is Molly’s email…
 
McDonnell, Molly Molly.McDonnell@mail.house.gov
 
Sincerely,
Steven Stefano
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Nice work Steve!
Comment left by Rick Bedlack on 01/07/2015
My uncle was in a clinical trial and he was really interested in the results. He is glad he got that results because he felt like if he didn't get any then to him it wouldn't have been as meaningful. He was able to find out if the drug that he tasted would be successful or not. He thought that was really nice to know. http://acr-research.com
Comment left by Travis Simons on 02/10/2015
Speaking at the Cherry Hill, NJ Support Group
Posted by: Kathy Delaney Thomas August 26, 2014
Kathy Delaney ThomasLasKMT Clinical Research Presentation Overview slidemonth, Karen Delaney Shideleff and I gave our ALS Clinical Research presentation to the Cherry Hill, NJ support group. We were grateful to be given the opportunity to share information about ALS clinical research with PALS and encourage them to evaluate whether participating in research is right for them.

We both find it very rewarding to be able to educate PALS about this important topic and are particularly inspired when we receive feedback like this:

[Two weeks after our presentation] "Another gentleman came in [to clinic]  all-systems-go for some studies he'd been ambivalent to join."
 
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Withdrawals from moderate doses of venafalaxine and 5htp, followed by a supervised dose of halidol and lithium, and a regiment of cold showers coupled with talk therapy have helped me to regain what I was unaware of having lost.
Comment left by Jordan Cutler on 09/08/2014
Great work Kathy! I am very proud of your work as an ALS Research Ambassador!!
Comment left by Rick Bedlack on 09/28/2014
PALS, Advocates and NEALS Research Ambassadors Storm the Hill
Posted by: Karen DiGiacomo June 03, 2014
Karen DiGiacomoRecently, PALS, advocates and our very own NEALS Research Ambassadors descended on Capitol Hill to educate our Senators and Representatives on this devastating disease called ALS.  One by one telling the most personal, emotional, heartbreaking stories to policymakers in the hopes that we will one day fund the means to a cure.

     

The following comments have been received in response to this blog post. To leave your own comments, please click here.
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Comment left by artem on 04/05/2015
State of North Carolina AOC Judicial Newsletter
Posted by: Karen DiGiacomo June 03, 2014
Karen DiGiacomoTEAM DiGiacomo featured in the building community section of the May newsletter - the judicial department. 
http://www1.aoc.state.nc.us/bb/bb20140505gd.htm
To leave a comment, please click here.
Gettysburg College hosts 'HOOPS for ALS'
Posted by: Karen DiGiacomo June 03, 2014
Karen DiGiacomohttp://www.eveningsun.com/local/ci_25471130/basketball-tournament-at-gettysburg-college-raises-money-als

To leave a comment, please click here.
TV Interview Taped for ALS Awareness Month
Posted by: Kathy Delaney Thomas April 24, 2014
Kathy Delaney ThomasKaren (Shideleff) and I taped an interview for a Philadelphia-based public affairs program, In Focus with Steve Highsmith, on WPHL-17. It will be shown in May for ALS Awareness Month. 
To leave a comment, please click here.
Spring 2013 Clinical Trial Update Webinar Synopsis
Posted by: ALS Trial Liaison March 14, 2014
ALS Trial Liaison NEALS Co-Chair Merit Cudkowicz’s latest presentation on ALS clinical trials emphasized the growing number of clinical studies and how this research is informing what we know about ALS.  There has been an explosion of ALS research in the last 30 years.  In 2012 there were approximately 1,400 papers published on ALS compared with 865 in 2008 and 426 in 2004.  Scientists now know more than 25 genes that can cause, or exacerbate, ALS or motor neuron disease.  Having this information is extremely hopeful for patients with ALS and their families; every time scientists learn about a new gene change that can cause motor neuron disease, they learn about a potential new therapy target.  As the lines between familial ALS (FALS) and sporadic ALS (SALS) become more blurred (7-10% of SALS have genetic mutation, mostly C9orf72), finding genes that play a role in the disease means more discoveries in the ALS field for both FALS and SALS.

The Complex Biology of ALS
ALS scientists are learning more about the causes of ALS.  There is a shifting mindset that ALS is likely not one disease, as the illness is very heterogeneous. Understanding this heterogeneity will guide treatment development in a more cohort focused manner.  The biggest obstacle to new therapy development is the complex biology of ALS.  While the ALS community has learned a great deal about this biology, the more we learn, the more apparent it is that this is a multisystem disorder and that many different pathways are involved. It is becoming clear that different patients with ALS exhibit different biology. Therefore, more understanding about the biology occurring in patients is critical. The tools to study the disease in people are becoming available now including iPS cells, advanced imagining and blood and cerebrospinal fluid studies. Even with present challenges, Dr. Cudkowicz is optimistic that new information on the biology (and new therapy targets) and new methods to study the disease in people will lead to much better therapies. The field can overcome it.  

How is the Field Taking Shape: Models in the Lab to Tools to Study People
There is a recognized need to impede delay in diagnosis.  A person with ALS sees an average of three specialists before receiving a diagnosis, or about twelve months.  The ALS field is developing the “red flag initiative” a campaign targeting and educating doctors by providing them list of top symptoms of ALS to ensure referral directly to neurologists.  Imaging technology has improved greatly in recent months and years allowing better study of disease targets in people.  Imaging can pick up abnormities, for instance in ALS in the brain and spinal cord, and can make sense of how certain drugs may change those abnormalities.  IPS technology is a way of understanding disease heterogeneity and developing more targeted treatments.  Scientists can take skin cells from ALS patients and dedifferentiate, or turn skin cell back into stem cell it came from, and make any cell of the body from that stem cell, particularly make a motor neuron.  This is a way of studying an individual disease.

The importance of biomarkers
There is currently a lack of ALS biomarkers, or markers of disease or response to therapy that might be used to diagnose ALS, observe disease progression, or monitor treatment effectiveness.  A NEALS biomarker study collects blood samples and cerebrospinal fluid (CSF) from patients with ALS with the goal of finding unique biological markers to treat the disease.  Once ALS biomarkers are discovered, therapy development for ALS will likely increase drastically.  

Clinical Trials in ALS
There are many trials in ALS, many with different study focuses and goals.  Some are looking at investigational drugs, others are looking at drugs approved in other disorders.  

Please find more information about each of the trial discussed in Dr. Cudkowicz’s webinar presentation:

Brainstorm
Tirasemtiv
Gilenya
Mexiletine - trial description page coming soon!
Nuedexta
GSK

What can you do?
To watch the webinar in its entirety and listen to the Q&A session, go here.

To read the remaining Q&A from the webinar go here.

Participate in clinical trials

Become clinical research advocate

Donate Samples

Any questions on clinical trials and the NEALS website can be directed to the ALS Clinical Trial Liaison: (877) 458-0631 or alstrials@partners.org



The following comments have been received in response to this blog post. To leave your own comments, please click here.
Do you guys conduct any clinical trials in Salt Lake City, UT? I was hoping to participate in something close sometime soon.
Comment left by Bill Shields on 04/23/2013
Hi Bill, There are currently no ongoing ALS clinical trials in Salt Lake City. However there is a NEALS member/ALS specialist located there. If you are interested, you could contact them to see if they have any ongoing sample collection studies. Here is the contact info: University of Utah Mark Bromberg, MD 50 North Medical Drive East Salt Lake City, Utah 84132 Phone: 810-585-7575 Please let me know if there is any other information I can provide for you!
Comment left by ALS Trial Liaison on 04/23/2013
Where can I find similar information focused on the West Coast and/or California?
Comment left by Peter S on 05/17/2013
WE ARE A TEAM
Posted by: Karen DiGiacomo February 25, 2014
Karen DiGiacomoI'd like you to introduce TEAM DiGiacomo to you.  A TEAM that was formed the very day my son, Louis  DiGiacomo, was diagnosed with ALS.  August 20, 2011, Louis proposed to his fiancé, Nicole.  Excited to begin his life and on top of the world, he never saw this coming.  On August 23, 2011, he was diagnosed with ALS.  And yet life was not finished, life would deal them yet another devastating blow.  Three days after his diagnosis, the loss of their baby.  A baby who, for three short days, was their hope.  At 29 years of age, his world, our world shattered.
 
An ex-captain of his lacrosse team, he lives for sports.  His passion lacrosse and fishing but an all-around athlete in hockey and football as well. Falling, tripping, no longer able to play in his weekly hockey league and garbled speech were the first indicators of something not quite right. Forced to quit his job at the date of diagnosis, the TEAM immediately embarked on ALS awareness and searched for clinical trials to participate in.  Louis’ reaction to clinical trials was ‘Where do I sign?’ Understanding that some PALS are hesitant to participate in trials was not something we had considered.  Admittedly, participation would not necessarily help Louis with his progression, but at least he was doing something to change the course of ALS, to MAKE A DIFFERENCE. 
 
Louis is fighting this with everything he has.  While Louis’ first year was gut-wrenching, an unexpected wonderful blessing arrived just one year and nine months into his progression.  His hope and strength stemming from Nicole and their beautiful baby girl, whose name is Zoey Hope. /images/files/Louis and Zoey.jpg
 
As a mom, my ALS advocacy and awareness efforts stemmed from a need to do something for my son.  It was evident to me that we desperately needed a cure for ALS and we needed it fast.  My first reactions of fear and devastation turned to strength, determination and hope.  The advocacy has encouraged me and my children to do more and work harder.  After speaking before the FDA at the ALS Special Public Hearing, Louis’ nomination in a National Mobility Awareness Local Hero Contest, ALS advocacy in Washington to speak to our congressmen, and his participation in two clinical trials, the next step for us was CRLI.  On behalf of TEAM DiGiacomo, Lauren (my daughter) and I attended the 2013 Clinical Research and Learning Institute (CRLI) and hope to inspire others to advocate, to join the fight against ALS and to encourage participation in clinical trials. 
 
This past November, with the clinical research training still fresh in my mind, I spoke at the MDA ALS Harvest for Hope Annual Event in Buffalo, NY.  Louis, Nicole and my mom listened as I talked about how ALS has affected my family and where research was just two years ago when Louis was diagnosed.  In 2011, researchers had isolated one familial gene mutation, SOD1, that played a role in familial ALS and where we are today with 22 familial ALS gene mutations and 7 sporadic mutations.  I addressed Louis’ progression in his first year.  The loss of his ability to speak, to walk more than two steps with great difficulty and the loss of his arm movement.  I explained that Louis was moving faster than research and we needed greater participation in clinical trials, new trials with new ideas and more funding for research in order to battle this disease.  I encouraged the tearful audience to continue to support funding for ALS, and hoped that they would please return next year with three more friends. 
 
Our gratitude to Johnita Hairston of MDA Buffalo & Todd Herman, Divisional VP - MDA, whose interest and support of TEAM DiGiacomo is unending.  Looking forward to 2014 Events and Trials! 
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Keep up the great work Karen. You are making a huge positive difference!
Comment left by Rick Bedlack on 03/16/2014
Want to learn more about ALS and Clinical Trials?
Posted by: Mona Shahbazi February 20, 2014
In my experience as a nurse practitioner working in field of ALS, one of the most powerful tools that patients need and can carry is knowledge.  The more you know about the disease process, and research being done in the field, the greater your ability is to engage in trials and take an active role in care of your disease.  So get out and get involved, attend support groups, join NEALS webinars, ask your clinic team and attend ALS conferences.  There is something new everyday and the more you know, the better informed decisions you can make about your care.  

Here's an upcoming oppurtinity:

We are holding an upcoming conference in New York City, sponsored by the HSS ALS program and the ALS association of Greater New York, on Saturday March 22nd.  There is a live web stream available for those who can not make it to the conference.  There will be a number of lectures and workshops on ALS, and Familial disease given by well known physicians and international researchers.  If you are interested, call 212-606-1812 or use this link: https://cmetracker.net/HSS/Files/Brochures/1610148.pdf

talks include:
*The Role of SOD1 in ALS: Two Decades of Discoveries, Defects and Disputes
*
ALS Associated with C9orf72 Mutations: Why Is This Important to Both Familial and Sporadic Cases?
*Update on Clinical Trials
*Workshops: diaphragm pacing, botox, Stem cell therapy and genetic testing

The following comments have been received in response to this blog post. To leave your own comments, please click here.
This is a good idea. Perhaps we could do something like this with the ALS support group meetings?
Comment left by Karen DiGiacomo on 02/21/2014
This is a good idea. Perhaps we could do something like this with the ALS support group meetings?
Comment left by Karen DiGiacomo on 02/21/2014
How to Choose an ALS Clinical Trial
Posted by: Dick Reilein December 16, 2013
How to Choose an ALS Clinical Trial  Choosing a clinical trial is NOT trying to judge whether it is going to be a break-through new drug or procedure, NOR is it attempting to figure out whether it will be successful.  The evaluation process is to determine if you fit the clinical trial requirements and how comfortable you are with the trial procedures, objectives, location, and duration.  ___________________________ There are several good online sources for investigating ALS clinical trials:        > clinicaltrials.gov, maintained by the US National Institutes of Health (I prefer this site because it has a better advanced search tool and the format of the trial report is easier to understand.)       > alsconsortium.org/search.php, maintained by NEALS Both allow simple searches (ALS) or more advanced searches  (combinations of terms: ALS, Illinois, Northwestern, Phase II, Recruiting and more). it's best to try using both sites to see which you like better. Your criteria for choosing a trial may be different than another patient. These criteria are good to eliminate trials which don't fit you:     1) Condition(#1, footnote): Amyotrophic Lateral Sclerosis or ALS     2) Search Terms (#2): Bulbar or ALS or Respiration     2) Recruitment: Recruiting ( not Closed or Inactive)     3) Location: You may want a trial site near where you live. If there are             none, you can specify a distance you are willing to travel. A trial              sponsor may have several other sites to consider.     4) Phase(#3): 0, I, II, III, IV Either search engine takes some getting used to. Try different entires for Description and Search Terms until you find what works best for you. The body of the trial description gives all these details and more. Further below, you will find an EXAMPLE SEARCH to practice on. __________________ INCLUSION Let's say you have identified some trials that interest you. You meet the INCLUSION criteria and the you are not eliminated by the EXCLUSION criteria.  The first step is to thoroughly review the INCLUSION CRITERIA (sometimes labelled as ELIGIBILITY). This information is easy to find. Every description of a clinical trial must include a list of those items that make you eligible for the trial. You will learn quickly, for instance, that a certain clinical trial is looking for people with familial ALS, not sporadic. This will possibly eliminate you from that particular study. Try another one until you find one that fits you. ____________________ EXCLUSION Of course, there are EXCLUSION CRITERIA, too. These elements are another hurdle to overcome. They often cause volunteers to drop out. It seems sometimes, exclusion criteria are too strict. But, the investigators want to ensure a homogeneous population for the test. This often lowers the cost of the trial and the time to get results. ___________________ INFORMED CONSENT & TRIAL PROTOCOL The next step is to ask for and get a copy of the INFORMED CONSENT. You should get the INFORMED CONSENT DOCUMENT from the clinical trial Investigator or administrator. This document must be available to, reviewed and signed by every volunteer before taking part in the trial. (Sometimes the TRIAL PROTOCOL is also available if the trial clinic and pharmaceutical company agree that it does not disclose secret information, and a confidentiality agreement has not been signed.) These documents (Informed Consent, for sure) should be read and understood COMPLETELY  before signing up for the trial. Ask the clinicians to explain anything you don't understand. The INFORMED CONSENT is written in 8th grade English. EXAMINING THE INFORMED CONSENT Many people with ALS are anxious to seek treatment hoping it will resolve the condition. No matter how anxious or hopeful you are, no matter how much you trust your doctor, you should not sign the Informed  Consent without examining it carefully, getting all your questions answered to your satisfaction. Both you and the investigator may terminate your participation in the trial at any time. You might consider what would cause you to leave the trial prematurely. And, ask what the investigator would cause her/him to drop you from the study. How much would your health and physical condition need to deteriorate? How closely do you need to follow the procedures? Can you miss using the drug once, or take it an hour late? Some Informed Consent documents contain a section entitled  "Experimental Subject's Bill of Rights", that some states require by legislation. In others, it's incorporated into the body of the document. If at a any time you feel your rights have been violated, you should contact the investigator or the Institutional Review Board immediately. EXAMINING THE PROTOCOL DOCUMENT Because the Informed Consent often lacks crucial information, the savvy patient may attempt to get a copy of the trial's Protocol. It is a lengthy document and contains lots of technical jargon, with scientific details about every detail of the study: rationale for the study; chemical make-up of the drug; test tube results; animal study results; previous human results. It also includes the detailed conduct of the present study: how many and what kind of patients; detailed inclusion and exclusion criteria; what drugs will be administered in addition to the one being tested; how often will tests be run; what side effects are expected and contingencies. For patients willing to slog through terminology intended for scientists and doctors, you may find there are some negotiating requirements; or you may find there are things you want to monitor more closely; and, the prior history of the drug/procedure and why they think the next phase is promising. It is not always possible pry the Protocol loose from the investigator and their staff. Throughout the process, always be pleasant and polite, but be firm in your resolve to get answers. There are several reasons for this:  They may say it is too technical for you to understand; your answer might be: "I or a relative have become very knowledgeable on ALS and am willing to try understanding the terminology." It's a pain to make copies of a 70-100 page document; "I am glad to make and pay for the copying." The most serious is the pharmaceutical company requires a confidentiality agreement. You may not be able to counter this. But, always be ready to ask more questions, below: QUESTIONS TO ASK YOUR DOCTOR AND PRINCIPAL INVESTIGATOR (see APPENDIX listing for more information) What is the purpose of the trial? What is the theory of this treatment? How many people have had this experimental treatment? How have they responded to treatment? What kind of side effects have been noticed with this treatment? How many patients experienced dire effects? How severe were the side effects? If I have side effects, what help will I receive?  Can you put me in contact with other patients participating in the study? Will I be disqualified if I pursue alternative treatment like acupuncture? Suppose I am responding positively to this treatment. Can I continue receiving the treatment?  What are my chances of getting a placebo? Will the study be blinded? Who is reviewing the study? And, how often? Is this a multi center trial? Where are the other locations? How long is the study? And, how does it compare to others? QUESTIONS TO ASK THOSE ADMINISTERING THE TRIAL When will the trial start? How often do I need to come into the clinic for a review? What medical records do you need from my personal neurologist? Who is the sponsoring clinic? Who can I contact if I'm having trouble with the treatment? phone numbers, email addresses, fax number, mailing address. QUESTIONS TO ASK YOURSELF Given my present condition, how much risk do I want to take? Am I fully aware of all my alternatives?  What is the best case scenario? What is the worst case scenario? (Based in part on a fact sheet adapted from  "Cancer Clinical Trials: Experimental Treatments an How They Can Help You" by Robert Finn, 1999) ENCOURAGEMENT Keep a POSITIVE  attitude and approach to finding a trial that you like. Don't give up after a few attempts with negative results. If you don't find something TODAY, try again in a WEEK or in a MONTH. The database changes and is updated to reflect new trials and eliminate old ones.  Use TODAY's DATE as the Last Entered date to get THE MOST RECENT UPDATES.   If you are persistent and dedicated to your search you will find one. GOOD LUCK. BE A VOLUNTEER FOR IMPROVED ALS TREATMENT. THANK YOU. ___________________ APPENDIX http://www.ciscrp.org/professional/facts_pat.html This is a good source: Center for Study of Clinical Research & Participation. You can use their menu of information (down the left side of the web page) to get much more insight and education in clinical trials. __________________________________ FOOTNOTES: (#1) Conditions— Use this field to specify a disease, disorder, syndrome, illness, or injury that is being studied. In ClinicalTrials.gov, conditions may also include other health-related issues such as lifespan, quality of life, and health risks. (#2) Search Terms— Use this field to specify words or phrases related to the studies you want to find. Performs a general search of the study information, including title, brief description, conditions, interventions, and locations. This field is equal to the Basic Search box at the top of every page. (#3) Phase— Use this field to select the study phase you want to include in your search. You can select more than one study phase. If you do not select any study phase, all study phases, including Phase 0, will be included in your search results. Phases are Food and Drug Administration (FDA) categories for describing the study of a drug based on the study's characteristics, such as the objective and number of participants. There are five phases:         Phase 0: Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).        Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety. The goal is to find out what the drug's most frequent adverse events are and, often, how the drug is metabolized and excreted.       Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition). For example, participants receiving the drug may be compared with similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.       Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.       Phase 4: Studies occurring after FDA has approved a drug for marketing. These include postmarket requirement and commitment studies that are required of or agreed to by the sponsor. These studies gather additional information about a drug's safety, efficacy, or optimal use. _________________ EXAMPLE SEARCH Let's take an example. Using the clinicaltrials.gov site, I entered the following search criteria:     Recruiting | Interventional Studies | ALS | United States, Massachusetts | Massachusetts General Hospital | Phase 2, 3 | received on or after: 01/09/2013 | updated on or before: 11/11/2013 With the following results: Rank Status        Study ____.       _____.            ____ 1        Recruiting.   Immunosuppression in Amyotrophic Lateral Sclerosis (ALS) Condition: Amyotrophic Lateral Sclerosis (ALS) Interventions: Drug: Basiliximab;   Drug: Methylprednisolone;   Drug: Prednisone;   Drug: Tacrolimus;   Drug: Mycophenolate mofetil 2 Recruiting Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS) Condition: Amyotrophic Lateral Sclerosis (ALS) Intervention: Device: NeuRx® Diaphragm Pacing System™ (DPS) 3 Recruiting Gilenya in Amyotrophic Lateral Sclerosis (ALS) Condition: Amyotrophic Lateral Sclerosis Intervention: Drug: Gilenya 4 Recruiting GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS) Condition: Amyotrophic Lateral Sclerosis Intervention: Drug: GM604 5 Recruiting Mexiletine in Sporadic Amyotrophic Lateral Sclerosis (SALS) Condition: Sporadic Amyotrophic Lateral Sclerosis Interventions: Drug: Mexiletine;   Drug: Placebo Let's choose the first entry to see what we can find. Read through the following information to learn about the study: Immunosuppression in Amyotrophic Lateral Sclerosis (ALS) NIPALS2013 _________________________ This study is currently recruiting participants. Verified October 2013 by Emory University Sponsor: Emory University Collaborator: ALS Association Information provided by (Responsible Party): Jonathan D. Glass, M.D., Emory University ClinicalTrials.gov Identifier: NCT01884571 First received: June 19, 2013 Last updated: October 11, 2013 Last verified: October 2013 History of Changes   Purpose This is a multicenter, 15-month study evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale (Revised) (ALSFRS-R) score in 30 subjects with Amyotrophic Lateral Sclerosis (ALS). Condition Intervention Amyotrophic Lateral Sclerosis (ALS) Drug: Basiliximab Drug: Methylprednisolone Drug: Prednisone Drug: Tacrolimus Drug: Mycophenolate mofetil Phase 2 Study Type: Interventional Study Design: Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment Official Title: A Novel Immunosuppression Intervention for the Treatment of Amyotrophic Lateral Sclerosis (ALS) Resource links provided by NLM: Genetics Home Reference related topics: amyotrophic lateral sclerosis MedlinePlus related topics: Amyotrophic Lateral Sclerosis Steroids Drug Information available for: Prednisolone Prednisolone acetate Prednisone Methylprednisolone acetate Methylprednisolone Prednisolone sodium phosphate Prednisolone phosphate Prednisolone sodium succinate Methylprednisolone sodium succinate Mycophenolic acid Mycophenolate sodium Tacrolimus Mycophenolate mofetil hydrochloride Mycophenolate mofetil Basiliximab U.S. FDA Resources  Further study details as provided by Emory University: Primary Outcome Measures: _______________________ Change in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: Screening, Baseline Visits 1, 2, and 3, Day 1, Months 1, 2, 3, 4, 5, 6, 8 ,10 , and 12, and at the Final Safety Visit, if a subject discontinues study drug early. Telephone ALSFRS-R will be collected at Months 7, 9 and 11. ] [ Designated as safety issue: No ] The Revised ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score (best of 48) from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). A clinical response will be defined as a rate of change of ALSFRS-R of +6 points over 6 months (mean of +1 point per month), where typically patients with ALS have a decline in ALSFRS-R by an average of -1/month. Secondary Outcome Measures: __________________________ Change in Slow Vital Capacity (SVC) [ Time Frame: Screening, Baseline Visits 1, 2, and 3, Day 1, Months 1, 2, 3, 4, 5, 6, 8 ,10 , and 12, and at the Final Safety Visit, if a subject discontinues study drug early. ] [ Designated as safety issue: No ] Vital capacity (VC), percent of predicted normal, will be determined using the slow VC method. SVC measures the amount of air exhaled following a deep breath. For this test, participants will hold a mouthpiece in their mouth, breathe in deeply, and breathe out as much air as they can. The test will be done seated in a chair and then repeated while lying on an exam table at the Screening Visit. For all other visits, this test will be done with seated in a chair. This test will take 15-20 minutes. At the Screening visit, eligibility for Group A will be determined utilizing upright SVC. Change in Hand-Held Dynamometry (HHD) [ Time Frame: Baseline Visits 1, 2, and 3, Day 1, Months 1, 2, 3, 4, 5, 6, 8 ,10 , and 12, and at the Final Safety Visit, if a subject discontinues study drug early. ] [ Designated as safety issue: No ] Hand held dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. Six proximal muscle groups will be examined bilaterally in both upper and lower extremities (shoulder flexion, elbow flexion, elbow extension, hip flexion, knee flexion, and knee extension), all of which have been validated against maximum voluntary isometric contraction (MVIC) testing. In addition, wrist extension, first dorsal interosseous contraction and ankle dorsiflexion will be measured bilaterally; these muscles are often affected in Amyotrophic Lateral Sclerosis. Change in Cytokine levels in Cerebrospinal Fluid (CSF) [ Time Frame: Baseline Visit 2 and Months 2, 6 and 12. ] [ Designated as safety issue: No ] Lumbar punctures (LPs) will be done to collect cerebrospinal fluid in order to characterize markers of the participants immune system and further the understanding of the immune factors that contribute to disease progression in ALS Collection of peripheral blood mononuclear cells (PBMCs) in Blood [ Time Frame: Baseline Visit 2, Day 1, Months 1, 2, 4, 6, 8 and 12, and at the Final Safety Visit if a subject discontinues study drug early. ] [ Designated as safety issue: No ] Blood will be drawn in order to characterize markers of the participants immune system and further the understanding of the immune factors that contribute to disease progression in ALS. Change in Left Grip Strength [ Time Frame: Baseline Visits 1, 2, and 3, Day 1, Months 1, 2, 3, 4, 5, 6, 8 ,10 , and 12, and at the Final Safety Visit, if a subject discontinues study drug early. ] [ Designated as safety issue: No ] Left hand grip will be measured using a study approved dynamometer to test the maximum isometric strength of the hand and forearm muscles. Change in Right Grip Strength [ Time Frame: Baseline Visits 1, 2, and 3, Day 1, Months 1, 2, 3, 4, 5, 6, 8 ,10 , and 12, and at the Final Safety Visit, if a subject discontinues study drug early. ] [ Designated as safety issue: No ] Right Hand grip will be measured using a study approved dynamometer to test the maximum isometric strength of the hand and forearm muscles. Change in Antibody levels in Cerebrospinal Fluid (CSF) [ Time Frame: Baseline Visit 2 and Months 2, 6 and 12. ] [ Designated as safety issue: No ] Lumbar punctures (LPs) will be done to collect cerebrospinal fluid in order to characterize markers of the participants immune system and further the understanding of the immune factors that contribute to disease progression in ALS Collection of Ribonucleic Acid (RNA) in Blood [ Time Frame: Baseline Visit 2, Day 1, and Months 1, 2, 4, 6, 8 and 12, and at the Final Safety Visit if a subject discontinues study drug early ] [ Designated as safety issue: No ] Blood will be collected for ribonucleic acid (RNA). Estimated Enrollment: 30 Study Start Date: October 2013 Estimated Study Completion Date: August 2015 Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure) Arms Assigned Interventions Experimental: Immunosuppression Regiment Basiliximab Methylprednisolone Prednisone Tacrolimus Mycophenolate mofetil Drug: Basiliximab 20 mg, IV (in the vein) on day 1 and 4. Other Name: Simulect Drug: Methylprednisolone 125 mg, IV (in the vein) on day 1. Other Name: Solumedrol Drug: Prednisone 60 mg PO (by mouth) on days 2-7, 40 mg PO days 8-14, 20 mg PO days 15-21, and 10mg PO days 22-28. Drug: Tacrolimus 1-5 mg PO, BID (twice a day) days 2-180. Other Name: Prograf Drug: Mycophenolate mofetil 500 mg PO, BID days 2-7, 500 mg PO each morning and 1000 mg each night, days 8-14, 1000 mg PO BID days 15-180. Other Name: Cellcept Detailed Description: In an ongoing safety trial of neural stem cell injections into the spinal cord of patients with ALS at Emory University, Atlanta, Georgia, one patient has demonstrated clear improvement by objective clinical and electrophysiological measures, a finding that is unheard of in patients with ALS. This patient had an improvement in ALSFRS-R by 1.4 points per month. In 826 historical controls from the Northeast ALS Consortium (NEALS) and the Western ALS Consortium (WALS) database where ALSFRS-R was documented at 2 or more visits, there have been no patients that have shown improvement in ALSFRS as seen in this case. Additionally, 5 patients in the stem cell trial who were not on mechanical ventilators at the time of surgery seem to have very slow disease progression as compared to the expectation from current understanding of typical disease course. This observation raises consideration for a disease-modifying effect of the novel immunosuppression regimen used in this trial. Also, given that ALS is clinically an extraordinarily heterogeneous disease, the diagnosis of ALS may represent a group of phenotypically similar but pathogenically variable disorders. It is possible that there exists a subset of patients with an immune-responsive ALS subtype that has not been previously recognized. Recent studies have furthered the understanding of the immune mechanisms that contribute to ALS progression. Microglia and lymphocytes have both neurotoxic and neuroprotective functions depending on activation states and physiologic conditions within the nervous system. Therefore, targeted immunotherapies that proportionally suppress neurotoxic immune elements, while sparing or promoting protective elements, seemingly have more potential to modify disease course in ALS than previously tested regimens. It is postulated that the immunosuppression treatment given to the stem cell patients may have exhibited neuroprotective effects by favorably promoting the ratio of regulatory T cells and other protective immune mediators in relation to neurotoxic immune modulators. It is hoped that this trial will optimize the chance of replicating these findings and allow the learning more about the complex changes that occur within the immune system in patients with ALS before and after treatment with an immunosuppression regimen. The primary objective of this study is to assess the clinical response rate of a novel immunosuppression regimen in a subset of ALS patients. The primary outcome measure will be rate of change of ALSFRS-R. A clinical response will be defined as a rate of change of ALSFRS-R of +6 points over a 6 month period (mean of change of +1 point per month). Secondary outcome measures will include slow vital capacity (SVC), grip strength, and hand held dynamometry (HHD). The change in rate of progression in clinical measures will be monitored to look for a potential disease-modifying effect of the immunosuppression regimen. Blood and cerebrospinal fluid immune system markers will be also be studied.   Eligibility _________ Ages Eligible for Study:   18 Years and older Genders Eligible for Study:   Both Accepts Healthy Volunteers:   No Criteria Group A Inclusion Criteria: Male or female patients 18-55 years of age. ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial Criteria. Symptom onset ≤ 24 months from screening visit. A score of ≥38 on the Revised ALS Functional Rating Scale. Slow vital capacity (SVC) measure >80% of predicted for gender, height and age at screening. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to the screening visit (riluzole-naïve subjects are permitted in the study). Negative tuberculosis (TB) test within 6 months of Day 1. Subjects medically able to undergo lumbar puncture (LP) as determined by the investigator (i.e., no bleeding disorder, allergy to local anesthetics, or a skin infection at or near the LP site). Capable of providing informed consent and following study procedures. Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study. Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating. Geographic accessibility to the study site. Group B Inclusion Criteria Male or female patients age 18 or older. ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial Criteria. Symptom onset >24 months from screening visit. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to the screening visit (riluzole-naïve subjects are permitted in the study). Negative tuberculosis (TB) test within 6 months of Day 1. Subjects medically able to undergo lumbar puncture (LP) as determined by the investigator (i.e., no bleeding disorder, allergy to local anesthetics, or a skin infection at or near the LP site). Capable of providing informed consent and following study procedures. Geographic accessibility to the study site. Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study. Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating. Group A & B Exclusion Criteria _________________________ Prior use of basiliximab, solumedrol, prednisone, tacrolimus or mycophenolate mofetil within 30 days of the Baseline Visit 1. Known allergy or sensitivity to basiliximab, solumedrol, prednisone, tacrolimus or mycophenolate mofetil or a formulation of one of these drugs. Treatment with an immunosuppressant medication within 30 days of the Baseline Visit. Active peptic ulcer disease. Any medical disorder that would make immunosuppression contraindicated including, but not limited to, human immunodeficiency virus (HIV), tuberculosis, or evidence of active cytomegalovirus (CMV) or infection. Subjects who have a diaphragm pacing system (DPS). Women who are pregnant, breastfeeding, or planning to become pregnant in the next 12 months. Active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements. Use of invasive or non-invasive mechanical ventilation (including Continuous Positive Airway Pressure (CPAP) or Bi-level Positive Airway Pressure (BiPAP)) for any part of the day or night prior to the Screening Visit (Group A only). Exposure to any other agent currently under investigation for the treatment of patients with ALS (off-label use or investigational) within 30 days of the Baseline Visit. Inability to safely complete study activities based on the discretion of the site investigator.   Contacts and Locations Please refer to this study by its ClinicalTrials.gov identifier: NCT01884571 Contacts Contact: Angela S Knox, MS, PMP 617-724-3314 aknox1@partners.org Contact: Daniela L Grasso 617-726-0842 dgrasso@partners.org Locations *United States, Georgia Emory University Recruiting Atlanta, Georgia, United States, 30322 Contact: Jane Bordeau, RN     404-727-1679     jrbord@emory.edu     Contact: Meraida Polak, RN     404-778-3807     mpolak@emory.edu     Principal Investigator: Jonathan D Glass, MD             Principal Investigator: Christina N Fournier, MD             *United States, Massachusetts Massachusetts General Hospital Recruiting Boston, Massachusetts, United States, 02114 Contact: Owen O'Connor     617-726-5059     ojoconnor@partners.org     Principal Investigator: James D Berry, MD             University of Massachusetts Medical School Not yet recruiting Worcester, Massachusetts, United States, 01655 Contact: Diane McKenna-Yasek, RN, BSN     508-856-4697     Diane.McKenna-Yasek@umassmed.edu     Principal Investigator: Robert Brown, MD, PhD             Sub-Investigator: Colin C Quinn, MD             *Sponsors and Collaborators Emory University ALS Association Investigators Principal Investigator: Jonathan D Glass, MD Emory University Principal Investigator: Christina N Fournier, MD Emory University   More Information Additional Information: Northeast ALS Consortium Website    No publications provided  Responsible Party: Jonathan D. Glass, M.D., Director, Emory ALS Clinic, Emory University ClinicalTrials.gov Identifier: NCT01884571     History of Changes Other Study ID Numbers: NIP-ALS-2013 Study First Received: June 19, 2013 Last Updated: October 11, 2013 Health Authority: United States: Institutional Review Board United States: Data and Safety Monitoring Board Keywords provided by Emory University: Immunosuppression ALS Additional relevant MeSH terms: Amyotrophic Lateral Sclerosis Sclerosis Motor Neuron Disease Spinal Cord Diseases Central Nervous System Diseases Nervous System Diseases Neurodegenerative Diseases TDP-43 Proteinopathies Neuromuscular Diseases Proteostasis Deficiencies Metabolic Diseases Pathologic Processes Methylprednisolone acetate Prednisolone acetate Methylprednisolone Methylprednisolone Hemisuccinate Prednisolone Prednisone Prednisolone hemisuccinate Prednisolone phosphate Mycophenolic Acid Mycophenolate mofetil Tacrolimus Basiliximab Anti-Inflammatory Agents Therapeutic Uses Pharmacologic Actions Antiemetics Autonomic Agents Peripheral Nervous System Agents ____________________
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Excellent post Dick. I hope others find this helpful as they decide whether or not to be in trials and if so which one to pick!
Comment left by Rick Bedlack on 12/12/2013
Thank you for this comprehensive report. Knowing why a participant might not be the right match for a particular study is a valuable contribution to matching studies with potential subjects.
Comment left by Marylee MacDonald on 09/26/2014
You want me to do what?
Posted by: Frank Heatwole November 18, 2013
That is the question I thought to myself during the NEALS PALS Clinical Research Learning Institute that I was invited to attend in beautiful Clearwater, Florida. I remember the day my doctor called and said "Frank, how would and your wife like to go to an ALS Conference? It would be a great weekend getaway. And you might learn something while you are there." Sounded good to me. I might learn something? I was so overwhelmed the first day I thought I should have stayed home. By the end of the second day I knew why I was there. They want me to be an ALS Research Ambassador. WOW. This Learning Institute has changed the way I look at ALS. Not for myself but for the future. The researcher, physicians, scientist, all the people dedicating their lives to ALS research need our help. Enrollment in ALS research studies is low. I was not aware of this, are you? I was diagnosed with ALS December 2011. I went for a 2nd opinion at Mayo Clinic, definitely ALS very slow progression. I was offered a clinical trial that was invasive and I choose not participate. Looking back now I was thinking of myself not the future generations. Now I think about the all the kids I coached (21 years)at the ballpark, my kids, my grandkids, how many of them will one day received a diagnosis of ALS themselves or someone they love. Will there be a cure? Will they know what caused it? How can the people dedicated to ALS research help us find the answers if we are not willing to help them. Think about it. I have.
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Frank, thanks for this excellent post. We are glad to have you on board as a research ambassador!
Comment left by Rick Bedlack on 12/12/2013
Clinical Research Presentation at Lehigh Valley Resource Group Meeting
Posted by: Kathy Delaney Thomas October 31, 2013
Kathy Delaney ThomasLast week, my sister Karen and I gave our first presentation as Research Ambassadors at the Lehigh Valley ALS support group meeting. We spoke to PALS and their caregivers for nearly 1.5 hours about ALS clinical research.
 
The topics we covered were:
Why isn’t there a cure for ALS?
What is clinical research?
Common questions about trials
Should I participate in research?
How can I get involved?
 
Attendance at the meeting was great – over 40 people – and what we had to say was well received.  The group asked many questions, with the resounding question being, “Why haven’t we known anything about this [low enrollment in trials]?”

We hope by being there we promoted clinical trial awareness, and encouraged those present to consider participating in clinical research.

Thank you to the Lehigh Valley group for allowing us to speak at your meeting!
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Kathy, I am so proud of you. Your presentation sounds like it was awesome. Keep up the great work as a research ambassador!
Comment left by Rick Bedlack on 12/12/2013
THE MORE THEY KNOW THE MORE THEY WILL KNOW
Posted by: David Ihde August 19, 2013
David Ihde

Was on vacation at OC Md for a week but kept on working; always wearing my "Shady McCoy" Eagles running back gifted T shirt from his event raising funds and awareness for us. Sporting it with my new power chair from the VA. Beach, SeaCrets club, Beatles Tribute Band, Elvis Tribute guy, dining out, reclining at pool, cruising the boardwalk (it wouldn't go under :) Wearing my Air Force hat and promoting knowledge and awareness to everybody that came up to thank me for my service or just to say they loved my purple power chair.
https://www.facebook.com/dave.ihde.1/media_set?set=a.10201079714692626.1073741841.1161224429&type=3 
Check photos os getting Vitamin D the easy way here at my Facebbok page.
Anywho, 2 things in the works; i was accepted for the Mexiletine Trial being conducted at Hershey medical center under the guidence of Dr. Zachery Simmons, that should start soon. Also this Thursday am Meeting with congressman Joe Pitts of PA to drum up support for the MODDERN Cures Act. Life is good, get out there and help make it last longer.
P.S. Attended 62 birthday party for fellow PALS, interviewed there with Pottstown Patch, an internet newspaper, check it out.

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David, you are an inspiration to us all. Great job enrolling in the Mexiletine Trial and meeting with congress!
Comment left by Rick Bedlack on 08/22/2013
Awesome job Dave! I am so glad you are able to participate in the Mexiletine Study. I hope you get as much out of the experience as I have in my clinical trials. Keep up the good work!!!
Comment left by Karen Shideleff on 09/03/2013
It's so easy!
Posted by: Karen Delaney Shideleff August 12, 2013
Karen Delaney ShideleffIt is nearly impossible to find anything "easy" with ALS. Every action takes thought and exertioN. This past week I finally found something that is EASY! I registered to be a donor for the National ALS Biorepository Study. It took a few emails, one phone call and a signature to join. If you are a member of the National ALS Registry, and all ALS patients should be, you can participate in this study! After learning about the study and reviewing the consent, I was able to set up my tissue donation appointment. I don't even have to leave my house, they come to me! Seriously, it doesn't get any easier. Study participants are seen in their home by a nurse who will draw blood, clip hair and nail specimens. You are also asked to give a urine sample. That's it! The Agency for Toxic Substances and Disease Registry is studying the best way to collect, store and share samples with ALS researchers. ATSDR is looking for 300 participants and they are barely at 100. There are thousands of ALS patients in America, with your help we should be able to reach 300 participants in no time at all. My pALS friends have signed up already. Email, call and sign up TODAY! Tell all your ALS friends. Lets break through the barrier of only 10% of ALS patients participating in ALS Research. All the information you need to get started is listed below!!! Information about the study- http://wwwn.cdc.gov/als/ALSBioRegistry.aspx Laurie Wagner, Pilot Study Coordinator can be reached by email- lwagner@mcking.com or phone 1-855-874-6912. Please sign up today and help ADVANCE ALS RESEARCH!
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Karen, thanks so much for this nice piece, and let's hope it helps stimulate enrollment in the Biorepository. Keep up the great advocacy work!
Comment left by Rick Bedlack on 08/22/2013
You could not be more right. I did the same a month or two ago, easy as pie, took about an hour in my home with Lucy reruns playing in the background. Everybody ought to be on this one. No pain lots of gain.
Comment left by Dave Ihde on 08/24/2013
Thank you for this important info. We MUST get involved to find a cure. My husband (PALS) will definitely participate. That simple , no reason not to!
Comment left by K/N Alvis on 10/12/2013
Trials are waiting for you...
Posted by: Karen Delaney Shideleff July 26, 2013
Karen Delaney ShideleffI was diagnosed with fALS on February, 18, 2011. Knowing I had a 50% chance of developing ALS, I vowed that I would do everything in my power to help advance ALS research. My mother and grandfather both had ALS & since my diagnosis my family has found out we carry the SOD1 mutation. I have participated in the ISIS trial, and currently I am enrolled in the Pyrimethamine trial. I have also donated blood, spinal fluid, & tissue to various studies. Less than 10% of ALS patients participate in trials, I want to change that by sharing my experiences with you. First, and most importantly, participating in studies and trials has given me a very powerful feeling of fighting back against a powerless disease. The psychological and emotional benefit of participating has been fantastic. I am fighting to save my family, your family and countless others and that benefit is immeasurable. Secondly, I have found that it takes time and dedication to participate in drug trials. I don't see this as a hardship as the benefits far outweigh the negatives. Many trials have compensation for travel and other expenses. If you don't feel as though you have the time to spare for a drug trial, consider participating in a clinical study. Often times it is just offering information about yourself and disease progression. You may be asked to give blood or tissue samples. Typically these samples cause little to no discomfort to the patient. And honestly, we can survive a needle stick, but we can't survive ALS! Lastly, I now fully understand why it takes so long to develop a medication and take it through the trial process. I have first hand experience in seeing the passion and dedication our researches have. If you want to see an advancement in research and a cure for this horrible disease, do something about it. There are tons of trials just waiting for you! Check out the trials posted on this website, look at ALSA.org, and search clinicaltrials.gov. All it takes to be a part of the fight against ALS is a little bit of time, lots of determination to finding a cure, and YOU!
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I was diagnosed with PMA last September, and am looking for the opportunities in related trials. Please keep me informed the coming trials. Thanks!
Comment left by Shaofu Wu on 07/27/2013
Nice article Karen. I am glad you are seeing first-hand the benefits of participating in ALS research, and especially happy to see you writing about your experience to inspire others. Thanks for all you are doing to help us beat ALS!
Comment left by Rick Bedlack on 08/12/2013
Well said Karen, everyone must do all they can for the benefit of all, Push On to Move Forward. Victory is in our grasp as long as we have hands to clasp.
Comment left by Dave Ihde on 08/24/2013
I would be very interested in participating in the trial since I was diagnosed march 2013.
Comment left by Robert Ferrara on 09/01/2013
LEBANON PA Veterans Hospital
Posted by: David Ihde July 24, 2013
David IhdeHere's  a letter I sent to Lebanon VA chief of neurology. Most of the Hershey vets go to the Lebanon VA for much of their medical needs. Not so much for ALS disease control, treatment and research though. However they have been outstanding in providing all the symptom assist items such as medication, equipment, home modification etc. I would though hope to be able to steer them to be more "hands on" in the fight and search for a cure. Not being critical just trying to get more people out there looking. I have met very few doctors that are up to date on ALS or that take an interest in being part of the solution, mostly just following the crowd and waiting for something to break out. And maybe that is all they can do but I'm gonna fan the fire anyway.
Dr.Julie Fieschko Keller                        
Chief Neurologist Lebanon Veterans Hospital
From: David F. Ihde, 144-50-8849 06/28/1955
Greetings,                                                                                                                                                           07/22/2013
I would like to present a proposal to your department at the Lebanon VA and perhaps you would be kind enough to pass this on to the appropriate Social Worker staff to also review. I am a 100% disability rated patient with ALS (Lou Gehrig’s Disease) and come to Lebanon for much of my care. Most of my ALS related care is done at Hershey medical center through Dr. Zachery Simmons and I’m sure you are aware there are many like me that come to you from Hershey. There are others also that don’t pursue care for their ALS by participating in clinic or support groups, I don’t know why perhaps they’ve given up hope but they must have their own reason. In any case there are many veteran PALS, Person with ALS, living in the territory covered by the Lebanon VA. I would like to propose to you to consider initiating a VA hospital based support group for us.
Such a group could only benefit all parties involved with this horrible disease. The Department of Defense which has declared this a service related disease and therefore pays out so much for our disability in support, treating symptoms and research. You will find that veteran PALS are the first to volunteer for research and a group like this would perhaps increase that participation through their gain of factual information about ongoing research. Also the D.O.D would have a group in one place where they could explain their needs and what we can do to help them.
The VA will benefit in organization and consolidation of information and program opportunities by enhancing their avenue of individual information by presenting it to a group. The doctors and therapists may be inclined to be more involved with their own pursuit of research into the latest developments and what’s on the horizon in ALS treatment. Perhaps even becoming part of the solution through new ideas and leading the pack. Their access to information and explanation to PALS will again improve participation, taking part in their own care and give a most needed commodity of hope and possibilities.
PALS of similar backgrounds will have a regular gathering place, somewhere to be comfortable among their peers perhaps drawing in those which previously have chosen not to participate in their own care. The possibility of being involved with research to a cure will increase. Research is not just clinical trials but also participating in clinics and support groups, joining the national and VA registry, taking part in surveys, joining in webinars, being part of the bio-repository, keeping others informed  and so on, the more they know the more they will know. You could arrange for guests to attend from the neurology department and other treatment or social offices and D.O.D, select a regular day and time per month to gather and make available the information discussed to those unable to attend. Knowledge is power and your increased participation in the ALS puzzle can only be good. Thank you.
Sincerely,
David F. Ihde 399 W. Pine St., Ephrata PA 17522 ph717-725-1968, email ihde2@ptd.net, @DAVIDIHDE
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David, I am the spouse of a recently-retired Army man who has now been diagnosed with ALS. We are trying to locate a neuropsychologist that takes TRICARE insurance to get a baseline early assessment. Just getting a genetic test counselor/MD that accepted TRICARE was challenging...eventually found one in the cancer category of TRICARE. Do you have any suggestions on how to find a neurophsychologist that takes TRICARE? Thank you!
Comment left by Maria Lund on 05/08/2014
DO U LIKE MY HAT?
Posted by: David Ihde July 19, 2013
David IhdeHere I am taking part in a study on brain-computer interfaces with Andrew Geronimo doctoral candidate at Center for Neural Engineering Penn State University. This took place at Hershey Medical Center, there will be two follow up appointments. In a nut shell brain-computer interfacing will allow those without physical capabilities to control many daily activities, needs and communication through eye sensing or imagined hand movement; A real plus to those suffering loss of physical ability. This was a simple harmless quick research procedure that anybody could participate in. This and many other research projects are on going, not all research is clinical trials, The more they know the more they will know, get involved please, help yourself by helping others. Push On to Move Forward.

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Great work! It is important to remind others that quality of life research is available to take part in. Another great way PALS, and healthy volunteers, can get involved! Thank you for all you do.
Comment left by ALS Trial Liaison on 07/19/2013
Help Yourself by Helping Others
Posted by: David Ihde July 13, 2013
David IhdeKeep on keepin' on. This week I had another article in the Ephrata Review, big color photo of me and Deb with O.J. Brigance during Advocacy day in D.C. a while back. Better late than never. The ephrata Review has been very supportive of the cause to bring awareness to the general public. This is I believe the 10th article in 12 weeks having something to do with ALS. Also sent one of my videos to Rep Bryan Cutler of PA as many of you know lost both mom and dad to ALS. And forwarded scan of article to those in association and shared with othe posting in facebook. Promote research and awareness each day, and by Aug 12 you will have made a difference 30 times, at least. http://www.youtube.com/watch?v=spm4kTqO9UU This video is about 10 seconds long, I think you have time to watch it.
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PUSH ON to MOVE FORWARD
Posted by: David Ihde July 10, 2013
David IhdeThis is a reply to one of my support group leaders about doing a presentaion on research participation. Group is once a month of course and the next open 15 minutes is November, if not then next winter or spring. Also filled out the PV survey today, did U?
Lets try November for now if possible but may have to go with a later date. I'd like to get the word out about the importance of PALS participating in research now if possible. It's not just clinical trials but also going to clinic, joining the national registry, joining the veterans registry, volunteering for the bio repository, telling people in decision making positions your story, coming to group, fund raising, contacting the local paper for an interview, being present at special events in the comunity representing the cause, filling out all those surveys that come via email, keeping aware of new developments, participating in webinars, find fact from fiction at Dr. Richard Bedlacks alsuntangled.org, join the NEALS blog etc. This is all part of research, the more they know the more they have to compare and a basis to work from. Nationwide less than 10% of PALS participate in research, that is an unbelievable number to me. Here in PA it is quite a bit higher and that is why we are in a good place if you gotta have it. Help yourself by helping others. I'm working on something of this nature to share with other PALS, for now I'll make a sort of handout which is quick. The only problem with handouts is they get stuffed in a folder and stay there. Doing something positive has its own placebo effect.
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C9 ID Project
Posted by: ALS Trial Liaison July 09, 2013
ALS Trial Liaison NEALS ALS Research Ambassador Steve Kolb was featured on a local television show, where he talked about his role as "C9 ID" Project Coordinator.  You can see the interview here: https://www.youtube.com/watch?feature=player_embedded&v=RfXl3ijqpHc#at=10 

Since taking on this role, Steve has given 11 presentations reaching over 200 people over multiple states.  As Steve describes, this project has really become his “second career.”  You can follow the project on Facebook at https://www.facebook.com/C9IDNOW. Terrific work Steve! 

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Steve, Why don't you have a fundraising link on the C9 Facebook page?
Comment left by Joe on 08/03/2013
ALL over the world
Posted by: David Ihde July 08, 2013
David IhdeThis is an inspiring facebook message I received from an old classmate and my reply to him. Spreading the word can only help.

Hi Dave,

I thought I would share this story with you.

I live in Chiang Mai in the north of Thailand, my son, now 16 goes to a Catholic school here--international program. As a Freshman he competed in the English Public Speaking Championships for Thailand. After 3 initial competitions which he won, he was competing for the Northern Thailand championship- Top 5 to compete for the National Championship. He was allowed to select the topic. He chose--Lou Gehrig - The Iron Man. He gave a background of the history of baseball (not a big sport here) and then the Yankees--and the life of Lou Gehrig and ALS. He pronounced the full name - Amyotrophic lateral sclerosis--and nailed it. He discussed the disease and its effects. He then showed the video clip of Lou Gehrig- "I am the luckiest man in the world" to end the speech. You could hear a pin drop. He won the competition.
A few months later I was at the school and the Principal came running across the parking lot. He said that speech was the most moving speech he had heard in his career in education. More importantly, he spread the awareness of ALS to a large group of people who never had heard of the disease before.

I see your posts and dedication and thought you might appreciate this story. Gus Reynolds
 
Thank you Gus, inspiration and positive comments are always welcome. I have recently finished training at the ALS Northeast center from Boston for research advocacy. I'll be doing a lot of talks and presentations trying to get other victims involved in research for a cure. I'd like to make this story a part of that presentation. It's a weird disease, you hardly ever hear of it but when you get it all of a sudden everybody seems to know another with it. Not many know it by ALS but most all know Lou Gehrig's disease. We're trying to bring awareness up to date so people don't assume it died with Lou. Thahnks for spreading the word.
Dave
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Hi Dave! Sounds like you've certainly been "on the move" raising awareness of ALS and advocating for a cure! Nice job! I attended the first NEALS CRLI in Clearwater, FL in the Fall of 2011. Since then I seize upon any occasion I can, to talk or present to audiences facts about ALS, and what it's like living with this disease. The way I look at it, I'm not only doing it for me, but speaking for all of my fellow PALS who are no longer able to speak. More power to you, and keep up the good work!
Comment left by Marilyn Sanford on 07/08/2013
KEEP IT UP!
Posted by: David Ihde July 03, 2013
David IhdeDay 2 after CRLI with ALS HOPE FOUNDATION at Valley Forge PA. Today informed Rep Mindy Fee and Gordon Denlinger of PA about CRLI and thanked them for supporting the 0,000 in budget for als.philadelphia chapter. Also sent photo of CRLI class. Also signed up for study on Brain Computer-Interfaces which will be conducted on July 17th at Hershey clinic. Contacted Dr. Fieschko at Lebanon VA about tele-medicine program possibilities. She called to tell me they all ready have it in place and operating. Not specifically for ALS patients but all her patients in the neurology department at their VA. Also this morning the third day put into works a presentaion for PALS at Hershey and Lancaster support groups. One downer, will miss an opportunity to attend visit by Congressman Dent at Hershey center and to represent veterans on PALS behalf due to prescheduled family annual vacation. You can't always get what you want, but if you try some time you just might find, you get what you need. Push On to Move Forward.
Dave Ihde-Ephrata PA
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CRLI VALLEY FORGE
Posted by: David Ihde July 02, 2013
David Ihdehttps://www.facebook.com/dave.ihde.1 This is a link to photo album from ALS HOPE FOUNDATION Clinical Research Learning Center held this past weekend in Valley Forge PA, much was learned and eaten :) To follow through on the objective of the training after a full two days; when I got home Sunday evening i immediately sorted and posted photo album on facebook. Shared it with all attendees I could contact, searched and found oher attendees and shared it with them. Next morning wrote a little synopsis of weekend and sent it with photo album link to ALS Association in D.C. director Jane Gilbert, ALS Philadelphia excecutive director Jim Piciotti, communications manager Tony Heyl, support group heads Judy Lyter and Joan Groh, doctors and VA Nuerologist. Then outlined a press release for local paper with photo. The Ephrata Review in PA is very supportive of the cause, they run something almost every week, I have 7 featured articles or photos over past few months. Then worked on presentation for support group meeting on participating and research, then joined this blog. That's it so far but it's only been a day and a half. Hopefully much more to come. My point to PALS is that research is not just applying for clinical trials, it is also filling out surveys, answering inquiries from the associations, becoming part of the national and VA registry, do the bio depository, go to support group. All these things provide information to others that can only help to advance the search for a cure. Help yourself by helping others. Press On to Move Forward.
Dave Ihde, Ephrata PA
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Thanks Dave! It was wonderful meeting you this past weekend. I am so glad to hear the experience of the Clinical Research Learning Institute was a positive one, and we look forward to hearing more from you on the NEALS blog!
Comment left by ALS Trial Liaison on 07/02/2013
You're REALLY on the move! Thanks for all of your hard work!
Comment left by Judy on 07/03/2013
Ken Johnson - ALS Research Advocacy Update
Posted by: Kenneth Johnson June 24, 2013
I guess it's about time I reported on what I have been up to in my advocacy efforts. My initial focus was to gain access to support groups, but eventually my efforts seemed naturally to branch out a bit in unexpected directions.

In April, my wife Rosemary and I went to The New Britain Hospital for Special Care, where most Connecticut PALS are treated, and met with the head of the neuromuscular division, Dr. Kevin Felice, as well as Bridgette Scalabrini (MSW) from the hospital and Stacey Rahl (MSW) from the  
Connecticut chapter of ALSA. Plans are presently underway to schedule presentations at the four support groups in Connecticut. We also discussed other roles Rosemary and I might play since most resources for PALS seem to be concentrated in the central and western portions of the state.

I have also had two meetings with the director of the Visiting Nurses Association of Southeastern Connecticut, Mary Lenzini, and she has invited me to address her staff regarding my own experience with clinical research as well as more general information regarding opportunities to participate in ALS clinical trials. Ms. Lenzini seems to think other VNA groups in the state might well be interested in having me speak, and she intends to use her contacts to investigate this opportunity to broaden the knowledge of nurses who sometimes care for PALS.

Hospice of Southeastern Connecticut has been advertising for some time their intention to broaden their mission to include chronically ill patients as well as those in the final stages of life.  Mary Lenzini has known the director of Hospice for years and plans to recommend my meeting with her staff as well for a presentation. I hope to have a meeting fairly soon.

In addition, I recently met with Dan Bacher, senior R&D engineer for BrainGate at Brown and founder of the Speak Your Mind Foundation (speakyourmindfoundation.org). The mission of the foundation is to provide volunteer technical assistance to ALS and other patients who are financially precluded from the benefits of more expensive technology. Dan seems truly committed to expanding the body of volunteers and the number of  patients who might be helped. I offered to provide any help I could to his admirable work.

I have an appointment with Dr. Cudkowicz at Massachusetts General Hospital this week, and I'll also be leaving behind some skin, blood, and CSF for the researchers.

I am beginning to see how multifaceted a good support system for PALS might be, and perhaps some of our work as advocates involves striving for a kind of seamlessness among the various resources available to patients. I would hope this would increase general awareness of the  
value of clinical research as well as the numbers of PALS willing to partake in clinical trials.



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Thank you Ken!
Comment left by ALS Trial Liaison on 06/24/2013
PALS Advocate Holly D. Ladd, JD
Posted by: ALS Trial Liaison June 07, 2013
ALS Trial Liaison Holly Ladd, JD is a standout ALS advocate, with a strong voice in the ALS community.  A lawyer by training, Holly has worked in the civil rights, international development and public health fields for 25 years, all the while gaining extensive experience with advocacy and organizing.  These skills have translated into her fight for increased awareness of ALS and ALS clinical trials, as well as the need for more financial resources. Holly recognizes the importance of clinical trial participation and has been enrolled in two biomarkers studies at Massachusetts General Hospital and the tirasemtiv phase IIB clinical trial.

As part of the ALS Association Advocacy Day and Public Policy Conference, Holly met with Senator Elizabeth Warren (D) of Massachusetts to urge her to co-sponsor the MODDERN Care Act.  This act would “accelerate the search for a treatment for ALS and other diseases by removing the barriers that limit medical innovation and by providing incentives to develop new treatments and diagnostic tools that can improve, prolong and, ultimately, save lives."  (ALS Assoc. website)  She also talked with Senator Warren about ways to remove road blocks that slow efforts to  a cure by getting stem cells, gene therapies, and small molecules into clinical testing faster.

What resonates most with Holly in carrying out her advocacy efforts are the lessons she learned as an HIV/AIDS advocate in the beginning of that epidemic “the two key elements in our success were advocacy and research, and we need to replicate that now for ALS.”

Thank you Holly!!


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Great job, Holly! Keep up the good work!
Comment left by Marilyn Sanford on 06/11/2013
Well done Holly. Keep up the great advocacy efforts!!
Comment left by Rick Bedlack on 06/15/2013
PALS Highlight
Posted by: ALS Trial Liaison May 07, 2013
ALS Trial Liaison Virginia Kolb and her husband Steve are enthusiastic, strong advocates in the fight against ALS.  A professional singer and actress, Virginia learned in December 2011 that she had ALS.  She eventually began having difficulty speaking.  With a great support system, Virginia and Steve began to educate themselves about the disease.  In doing so, they met Virginia’s neurologist Dr. Merit Cudkowicz and embarked upon finding clinical studies in which to participate.  In December, 2012, it was also discovered that Virginia had the C90rf72 mutation.  

More than willing to try an approach with a scientific basis, Virginia and Steve became participants in the biomarkers study.  As part of the study, researchers collect skin, blood, and cerebrospinal fluid (CSF) samples to learn more about the cause of ALS and to find unique biological markers.  “One of the great frustrations for people with ALS and their caregivers and family members is a sense of hopelessness.  Participation in clinical trials is our way of doing something,” they shared.  Virginia is also in the process of enrolling in the BENEFIT-ALS phase IIB trial of tirasemtiv (formerly CK-2017357); this means “I’m doing something that helps,” she states.  Steve adds, “Even for trials that are not successful, it is critical that we rule out those options…knowing keeps us moving forward.”

In addition to enrolling in clinical trials, Virginia and Steve attended the 2012 Clinical Research Learning Institute (CRLI) a training which prepared them for leading other people with ALS (PALS) and caregivers to follow in their footsteps.  The CRLI is an intensive program dedicated to educating PALS and caregivers about clinical research and therapy development while empowering them to become advocates for ALS clinical research.  “I felt honored to be there representing the ALS community.  With it comes a commitment to get out and make a difference on behalf of that community (in any way we can),” Steve revealed.  Virginia described her experience at the CRLI in one word: hope.  Since the CRLI, Steve has spoken at their support group, made numerous contacts in their community, and launched the initial phase of a C9orf72 research funding project.  

It is evident that the feelings that come with a devastating diagnosis have grown into inspiration and motivation for both Virginia and Steve.  When they discovered a fundraising need for research that will specifically target C9ORF72, they were immediately on board.  The project seeks to identify individuals and families with the C9ORF72 mutation to collect information on the natural history of this form of ALS. This information will be critical for development of new treatments for people with C9ORF72 ALS. It will also allow researchers to notify people with C9ORF72 about future investigational treatments that will target this type of ALS.

With a background in non-profit administration, Steve knew he wanted to use a fundraising model that would give potential donors the information they need about ALS and C9ORF72 to make an informed decision, “It is crucial to identify people whose hearts and mission align with your own.”  The plan entails holding a series of meetings located near NEALS research sites, thereby incorporating experts in the field, before everyone comes together at a large fundraising event.  “I am thrilled that the Kolbs are helping to get us that much closer to research into C9ORF72,” declares Merit Cudkowicz, NEALS Co-Chair and Neurology Chief at Massachusetts General Hospital.  “I cannot commend them enough.  Their valuable efforts will help so many.”  

Fundraising is currently in its preliminary phase.  Steve and Virginia are raising 25,000 dollars towards the final projected cost for the entire large-scale identification process.  After this phase, they know that, “it will feel great to convey to donors that ALL of their contributions will go directly to the research…that’s a powerful statement to be able to make.”  Social media has been an immensely valuable tool in reaching their goal.  In particular Facebook has allowed them to “connect with people in the ALS community, convey helpful information, and provide encouragement to other PALS and caregivers.” Some of the contributions coming into the project are from donors whom Steve and Virginia do not know.  When talking about the first donations they received, Steve said, “I saw contributions linked to a Facebook page of a person I didn’t even know, which was a page that was shared by someone else I didn’t know, who shared it from someone else…who we did not know!  We were three steps removed from someone else whose heart aligned with this project.”  Many of these contributors may not have been identified as donors by Steve and Virginia otherwise.  The Kolbs, many friends and family members, as well as these ‘strangers’ were able to connect because of social media.  

Virginia and Steve will not stop there.  Backed by the support of loving friends and family and a close tie with their ALS Multidisciplinary team, they poignantly declared, “There is still lots to do! The bottom line is, we are all working together for a cure…some things in life require us to work together.  Finding a cure has to be a team effort.  The bigger the team, the faster we will get there.”

See Virginia and Steve’s fundraising page here:
http://www.gofundm
e.com/1u0tpc




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As a participant in the 2011 CRLI , I welcome you aboard! Your energy, focus and expertise are inspiring and much needed! Thank you both for the extraordinary commitment you are making to this fund raising project for research on the C9orf72 mutation. Go for it! The answers are out there waiting to be found!
Comment left by Mary Murray on 03/08/2013
Way to go Virginia and Steve. Keep up the great advocacy work. Don't forget to log in your outreach efforts on the table above. See you out on the trail!
Comment left by Rick Bedlack on 04/16/2013
Springing Into Action
Posted by: Richard Bedlack, MD, PhD May 01, 2013
Richard Bedlack, MD, PhDIt has been a busy couple of months, but oh what fun!  

On Tuesday March 12, I participated in the first Duke Voices of Medicine storytelling show at the Casbah Club in downtown Durham NC.  What an incredible experience.  I was so moved by some of the stories I heard that night, and proud to tell one of my own about a patient who left a special mark on me.  If you want to listen to it, here is the link http://news.medicine.duke.edu/voices-of-medicine-stories/

On Saturday March 16, I attended the ALSA Walk in Fayetteville NC.  It was cool, damp and windy, which made my hair look even worse than usual, but we still had a great turnout.  I enjoyed walking alongside some of my patients and meeting their wonderful families and friends.  At the end I gave a brief research update.

On Monday April 8, I played in the Nancy’s Crew Spring ALS Golf Classic in Wake Forest NC.  It was a gorgeous sunny and warm day.  I had some great golf pants on (can you find them in the picture?), and my team even briefly had the lead; this is because we were first to finish and post our score.  When all the other teams came in after us with their scorecards and we learned that we actually came in last place.  Regardless, this was a great opportunity to teach several of the other golfers about ALS and to see PALS Nancy and her family and many friends.

 



Saturday April 13 brought the Triangle ALSA Walk in downtown Raleigh NC.  Another gorgeous day and a packed house with so many fabulous tee shirts made by different teams of PALS and their families.  Some of the PALS I walked alongside even gave me some great new music recommendations.  At the end of the event I got a chance to update this amazing group with my plans for expanding ALS care services at Duke and the Durham VAMC, and on some of the research we are doing as well.

 


The following day I travelled to Pinehurst NC (golf mecca!) to attend a Celebration of Life, put on by the family of a patient who lost his battle with ALS last year.   I heard some fantastic stories about him and got to listen to a big band play some of his favorite music.  I got to speak about ALS, and discuss the exciting progress being made toward understanding the causes and progression of the disease, biomarkers, treatments under development and the importance of enrollment in trials.

Recently I travelled to Wisconsin and Chicago.  The airlines tried to stop me by cancelling my first flight, then delaying me, then losing my luggage but none of it worked! I made it there in time to visit with my friend and colleague Professor Paul Barkhaus and to give Grand Rounds on ALSUntangled at Medical College of Wisconsin on April 19.  The next day I spoke to around 200 patients and families from Wisconsin, and the day after that I drove into Chicago and spoke to another 100 or so patients and families from that area.  Those talks highlighted alternative and off-label therapies, and the ALSUntangled program we developed to scientifically review them.  While I was on this trip I got a special new hat and tie…made from Wisconsin cheddar of course!



As I write this, I am packing for my next trip to Washington DC for National ALS Advocacy Days May 9-11.  I hope to see some of our NEALS Research Ambassadors there.  Maybe I’ll even wear my new hat and tie….

The following comments have been received in response to this blog post. To leave your own comments, please click here.
You have been busy. I saw your photo with Bob. We found each other on the forums and Facebook PALS page. He stops by to see me when he travels to DC. I was part of a meeting with the governor of VA. When he declared May as ALS awareness month in VA. http://www.flickr.com/photos/governorva/8705941954/in/set-72157633412349736/lightbox/ Sorry I will miss hearing you in DC. I will be at a concert with hubby and friends :) Keep up the Great work on Untangled !
Comment left by Lisa Davis on 05/09/2013
Advocacy Work
Posted by: Marilyn Sanford January 25, 2013
Since attending the first NEALS Consortium's Clinical Research Learning Institure, here are links to some of the work I have been doing to help raise awareness of the disease and the importance of ongoing clinical trials:

I participated in the Boston ALS Walk (SEPTEMBER 2012)
 
When the news of the failure of Dexpramipexole broke, I was interviewed by The Boston Globe and WBUR (an NPR radio station) as they wanted to get the reaction of PALS to the disappointing news.
Boston Globe article: Here
WBUR: Here (JANUARY 2013)
 
I presented, along with members of the Mass ALS Assoc., to about 200+ employees of Biogen Idec in Cambridge and Weston, on the topic "Living with ALS."  (JULY 2012)
 
I also delivered a similar presentation in Boston to a group of Federal agency managers, at the request of the Combined Health Charities (CHC), to raise awareness of ALS as part of the kick-off of the Federal employees' charitable giving campaign. (AUGUST 2012)


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Great work Marilyn!
Comment left by Rick Bedlack on 01/30/2013
In Remembrance of Laura Tuttle
Posted by: ALS Trial Liaison January 22, 2013
ALS Trial Liaison
It is with much sorrow that we must share the passing of a remarkable woman, Laura Tuttle.  An unwavering ALS advocate, Laura fought against ALS for three years.  

Upon receiving her diagnosis, she began her mission to help find a cure for ALS.  Without delay, she explored clinical trials and over the course of her disease participated in several clinical research studies available to her.  But, she did not stop there.  

Laura was and still is an advocate and crusader for PALS. She was an active participant in several ALS Association Advocacy Days as well as the 2011 NEALS/ALSRG ALS Clinical Research Learning Institute, a training dedicated to empowering PALS and caregivers to be advocates for ALS clinical research.  She taught the importance of enrollment in ALS research.  Because of Laura’s passion, compassion and teaching, Laura was spotlighted in an interview last spring for NEALS. There is no finer example of her fighting spirit than her quote from the article, “My philosophy is I would rather do something than nothing.  Nothing to me just means sitting back and letting the disease drive my life.”  She continued, “It is amazing the courage and energy that you get when you’re facing a terminal illness.”

Because of her committed efforts, Laura received the National ALS Heroes award from the ALS Association.

Her clinical care team at the Massachusetts General Hospital remembers her fondly.
“Because of Laura, I am a better nurse and a better advocate for PALS, and will always hold my journey with Laura close to my heart,” stated Darlene Sawicki, NP.  Michele Parkinson, RN, added, "Working with Laura and her family during Laura's fight against ALS was a true honor. Her compassionate and resilient spirit, as well as her wonderful sense of humor will be truly missed."


The following comments have been received in response to this blog post. To leave your own comments, please click here.
I first had the pleasure of meeting Laura and Rich at the NEALS Consortium's first Clinical Research Learning Institute held in Clearwater, FL in October 2011. Unfortunately I didn't get to see her again until ALS Night at Fenway last May, but have followed her various advocacy efforts on the part of ALS research. She was definitely a role model for all of us PALS, in that she did not allow the disease to "define" her, but showed her "amazing courage and energy" in fighting back on so many fronts! My deepest sympathies to Rich and the family. She will be sorely missed by the ALS community!
Comment left by Marilyn Sanford on 01/25/2013
Laura Tuttle
Posted by: Mary Murray January 22, 2013
It is with deep sympathy for her family and friends and great respect for her selfless dedication to ALS Advocacy, that I bear the sad news of Laura Tuttles passing last December. Laura is missed by so many of us as a staunch Ambassador, a thoughtful and caring advocate, and a warm and energetic presence in the continuing search for treatments for ALS. There is a more detailed account of Laura's contributions in this Blog that is worth re-reading as an inspiration for the rest of us.

I met both Laura and Rob Tison at the 2011 CRLI and also at the ALSA Advocacy Day in Washington, D.C. last May. It was a privilege to know them and I would urge us all to keep both Laura and Rob in our hearts and minds as we continue this quest for treatments for ALS.





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Thank you for your kind words Mary.
Comment left by ALS Trial Liaison on 01/22/2013
Biogen Announces Discontinued Development of Dexpramipexole
Posted by: Richard Bedlack, MD, PhD January 08, 2013
Richard Bedlack, MD, PhDThis past Thursday, Biogen Idec announced that it would be discontinuing development of dexpramipexole based on its negative phase III trial results.  While this is understandably disappointing to many there are some important positives to take away from this trial:

1. This was the most sophisticated, well-organized, efficient trial that has ever been run in ALS and it serves as a nice model for how future phase 3 trials can be run as partnerships between PALS, trial consortia and industry.

2. By allowing home visits, this trial shows that we can run trials that have less burden for PALS and still get good data.

3. This trial used a new and important end-point which combines both function and survival, and is likely to become the main endpoint for future ALS trials.

4. This trial showed that there are no short cuts to finding the right answer in this field.  We had great looking phase 2 data, and impressive anecdotal reports, and some were suggesting that should be enough to approve dex.  If we had stopped there, we would have large numbers of future PALS on a drug that was no better than a placebo.  While we still need to pay close attention to early phase trials and to anecdotal reports we cannot rest on them alone.

5. It is important to remember that we got a definitive answer to an important question here: Does dex work?  It wasn't the answer that we wanted but it was loud and clear.  The PALS and CALS who participated in the dex trials should be congratulated and be proud that they helped us answer this important question.  Every question we answer brings us one small step closer toward understanding ALS and having better therapies for it.  


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I think the largest challenge we have is that we are compilation of individuals not a disease. There may be as many as 18 different contributing factors and not every drug is going to work in every patient or perhaps even the majority of patients. Until we get patients categorized by onset, ethnicity, history, head injuries, pesticide exposure, prepubescent athleticism etc. they are going to have bad study results. I am not only a patient with ALS but I've worked in the industry for 30 years. Many of my medical devices went through clinical trials and I know difficult they can be. But the bottom line is we don't have a disease here, technically, we have a syndrome.
Comment left by Hollister Lindley on 01/08/2013
Thank you, thank you! As a participant in the DEX trial, I would like to thank all of the research teams who worked so hard and with such hope on this trial. The team headed by Dr. Nazem Atassi with his research team including Michael McKenery and Lindsay Kaplan at Massachusetts General Hospital where I was enrolled were an inspiration to me. My hope is that this will not discourage them and all the people involved in centers around the world from staying with the quest for treatments. A big THANK YOU to all who worked on and participated in the trial. And another big thank you to the people at Biogen Idec, several of whom I met both at their Cambridge lab and at ALS fundraiser walks in Boston. They turned out in large numbers to walk . They put more than hours into their work and have publically promised to stay with us in the ongoing search for treatments. Thank you all!
Comment left by Mary Murray on 01/22/2013
NEALS Research Ambassador Joins Prize4Life Board of Directors
Posted by: ALS Trial Liaison December 26, 2012
ALS Trial Liaison NEALS Research Ambassador Pete Frates has accepted an appointment to the Prize4Life Board of Directors.  Frates, who attended the 2012 Clinical Research Learning Institute, and his sister Ms. Jennifer Mayo join this team of outstanding ALS researchers and advocates.  In this role he will help to promote clinical research and acceleration of a treatment for ALS.

Prize4Life CEO Avi Kremer was recently quoted, “Both Pete and Jenn bring strong leadership and unique perspectives, as well as impressive track records of innovation and management to our team.  Their individual and combined contributions to raising awareness about the need for new treatments and a cure for ALS will, undoubtedly, help us to achieve this goal as soon as possible.”

NEALS would like to congratulate Pete on his new endeavor!

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Congratulations Pete. I am very proud of the work you are doing as a Research Ambassador!
Comment left by Rick Bedlack on 01/02/2013
Thanks to you both, Pete and Jenn, for contributing your time and talents in so many ways to finding treatments for ALS! Making ALS visible to many people who see you working for a cure is such an encouragement to all of us with ALS. Can't thank you enough!
Comment left by Mary Murray on 01/22/2013
Looking Forward to the Clinical Research Learning Institute!
Posted by: ALS Trial Liaison October 12, 2012
ALS Trial Liaison There are just two weeks until the Second Annual ALS Clinical Research Learning Institute (ALSCRLI) and NEALS researchers are ready and excited! The CRLI, sponsored by the ALS Association’s TREAT ALS initiative, is an intensive program dedicated to educate 10 patient-caregiver pairs on clinical research and to empower them to be more effective research advocates.  Drs.  Richard Bedlack, Merit Cudkowicz, Jeremy Shefner, Nazem Atassi, and Eric Macklin will present various topics highlighting the fundamentals of clinical research and providing tips for more effective advocacy, and will field questions from participants, who will become “NEALS Research Ambassadors.”  NEALS faculty will be joined by other leaders from pharma, foundation, and patient care organizations to facilitate thought-provoking discussions.  The training addresses questions and topics such as:

Why does the research take so long?
What rules must researchers follow?
Why do we really need placebos in clinical trials?
What can I do to advocate for clinical trials in ALS?

It is the hope of the NEALS faculty that everyone in attendance will not only benefit from the training but forge new relationships.  As one participant stated about last year’s event, “The CRLI was absolutely wonderful.  There was a lot of valuable information packed in…it was interesting to meet other patients and caregivers who have an outlook like mine (on clinical trial participation.)”   
This year, the CRLI will provide patient-caregiver pairs with take-a-way tools that they can use to share their knowledge from the training, thereby advocating for clinical trials, with their contacts, whether speaking at a support group or writing to their Congressman, amongst other actions they can take.  Along with last year’s Research Ambassadors, they will blog about their advocacy efforts, experiences, and connections on the NEALS website.  

A new feature will be added to the website: our advocacy tracker.  This tool will display all the outreach efforts they have collectively accomplished and will allow us to see the difference all our Research Ambassadors are truly making.

We welcome our new Research Ambassadors and we hope they enjoy their time at the CRLI just as much as we do!

Read about the 2011 CRLI


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My husband and I look forward to attending the research conference next week and enjoying the discussions, lectures and workshops on how to be better patient advocates. I strongly believe that educated, empowered PALS and CALS are vital to ALS research and development of effective treatments and the cure. As a NEALS Research Ambassador, I plan to educate and encourage others to become involved and work together toward this goal. I thank NEALS and the ALS Clinical Research Learning Institute for this wonderful and exciting opportunity and look forward to meeting all of you.
Comment left by Susan Speranza on 10/19/2012
Thank you for your post Susan. We are looking forward to meeting you later this week!
Comment left by ALS Trial Liaison on 10/22/2012
The Passing of Rob Tison
Posted by: ALS Trial Liaison September 19, 2012
ALS Trial Liaison It is with great sadness that we must share the passing of Rob Tison.  To say that Rob was an active advocate in the ALS community would be an understatement.  Since his diagnosis of ALS in March 2010, he touched so many lives, many of whom he never met.  Rob was a prominent figure on social media and online communities, where he could be found promoting ALS clinical trials and facilitating other PALS’ knowledge of the ALS research community.  He discussed his own clinical trial participation and did so in a comprehensive, understandable way.  Many PALS who had online exchanges with him have noted not only his undeterred drive towards finding a cure, but their own honor with having known such a man.

In October 2011, Rob attended the Clinical Research Learning Insitute, thereby NEALS naming him a Clinical Research Ambassador.  In this role, he educated others with ALS about clinical trials and encouraged them to enroll in such studies.   This past May at the ALS Association Advocacy Day in Washington DC, Rob was received the Rasmussen Advocate of the Year Award, which is presented to an individual whose personal efforts increase awareness of ALS and The ALS Association through education and advocacy at the local, state or federal level.

One of his most recent contributions to the PALS community was to devise and administer a survey assessing PALS’ awareness and perceptions of the CDC ALS Registry and from that to present to the CDC strategies for reach out to more PALS to join the Registry, a tool to collect, manage, and analyze data about people with ALS.

Rob’s family and friends have created a Facebook Page in his name: 
http://www.facebook.com/PerseveringYouAreAGameChanger



“Margaret Mead once said ‘Never doubt that a small group of thoughtful committed citizens can work together to make a difference.  In fact that is the only thing that ever does.’  Rob Tison’s efforts against ALS have been nothing short of inspirational, and have undoubtedly ‘made a difference’ in moving us closer to better treatments for this terrible disease.”
– Richard Bedlack, MD, PhD, Duke University

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Like many others, I was stunned to read that Rob Tison died on September 10. We met him at the CRLI in Florida last November and he was such a vital presence and energetic participant that it is hard for me to believe he is lost to us so soon. I did not realize at that time that he was the dynamic 'Persevering' I had been following in Patients Like Me and TDI forums where he wrote such inciteful analysis of current research - just look at his entries on this Blog. In May we met again in Washington, DC, at the ALSA Advocacy Day meetings. I realized he had not been feeling well, but I did not read the signs. It is another cruel reality of the ALS community that before we have time to appreciate a new friend, so often that friend is gone. My sincere sympathy goes to Rob's lovely wife, Kelly and their wonderful son and daughter.
Comment left by Mary Murray on 09/19/2012
Dr. Bedlack and my wife, Mary, have expressed for me what a privilege it was to have met Rob Tison and his young family. And I can't begin to imagine how many folks who never met him, and perhaps don't even know his name, who benefit so much from his highly intelligent, tireless, and effective advocacy. As I was picking up an order at a large food court one evening near the recent ALS Advocacy meeting HQ in DC, I saw Rob and Kelly and their son and daughter sitting together in a quiet corner sharing a meal. I did not go over. They looked so at ease and happy, gathered around a simple meal. I did not want to interrupt them. That's the memory I'll keep of Rob - husband, father, who worked so hard so that other PALS, CALS, and their children could have many more years together. Sincerest condolences, and gratitude, to the Tison family.
Comment left by Bob Murray on 09/20/2012
We miss Persevering online already. I had so hoped that he might see some action and deliverables related to his recommendations for the Registry before his death. Rob definitely was a special force. I hope that we will keep his memory alive by continuing to push and prod for improvements in the fight against ALS.
Comment left by R on 09/21/2012
Rob exhibited an unyielding quest to find answers to the ALS mystery. His dedication was constant and his keen insight was always thought provoking. The ALS community will miss his thoughtful questioning and probing into the research and discovery processes. My sympathy to his wife, Kelly and their children on his loss.
Comment left by Tom Kettler on 10/01/2012
PALS Highlight
Posted by: ALS Trial Liaison August 14, 2012
ALS Trial Liaison  Since learning in December 2009 that she had ALS, Laura has been a prominent clinical trial advocate and strong voice in the PALS community.  Shortly after her diagnosis, her decision to enroll in ALS clinical trials was an easy one.  “When I was first diagnosed with ALS, the very first thing I did was start researching clinical trials because, as an RN and former hospice nurse, I knew what the progression was going to be.”  She has been proudly enrolled in five ALS clinical trials. “My philosophy is I would rather do something than nothing.  Nothing to me just means sitting back and letting the disease drive my life.”

Laura’s experiences as a participant in clinical trials have been highly positive.  With each trial she has felt appreciated and valued.  While she acknowledges that some trials have more in-depth or lengthy procedures as part of their protocols which can require more time and energy, staff at her  local ALS Multidisciplinary Clinic continuously made her feel well-connected throughout the clinical trial process.  “They answer all my questions; the clinical trial team makes all the difference.”  It is  through this active participation and close contact with staff, that she has also learned more about the importance of observational trials, which she notes PALS should be careful not to rule out.  For many PALS, observational trials can be overlooked in lieu of interventional trials. For Laura, who has completed three observational trials and is considering her fourth, this is not the case.  “Knowing my blood will go to a repository that other researchers can use is huge for me because it is a rare disorder… we need more participants to get a better idea of what this disease is all about,” referring to her enrollment in a biomarkers trial, a study that involved sample collection for the purpose of learning more about diagnosis and disease progression.  

In talking with other PALS, Laura considers the number one concern PALS have with getting involved in a trial is the possibility of becoming randomized to a placebo arm.  Laura shares her outlook, “You need to have a broader view on the picture; to participate in an interventional trial that involves using a placebo, it may or may not be (directly) helping me but ultimately it is helping other PALS.”  It may help PALS who are considering clinical trials to know that in many trials if the treatment under investigation is shown to provide benefit, anyone enrolled (regardless of treatment arm) can continue to receive the treatment after the trial. “I know for myself that I would love something to slow my progression, but I wouldn’t know that it could do that if it hadn’t been thoroughly researched first; that’s why we have clinical trials.”

Laura’s can-do attitude carries over into her advocacy work.  ALS advocacy is another route she has undertaken and she encourages other PALS to do the same.  She has attended both the NEALS/ALSRG ALS Clinical Research Learning Institute (CRLI) and the ALS Association Advocacy Day.  The CRLI is an intensive program dedicated to educating PALS and their caregivers on clinical research and therapy development and empowering this group to be advocates for ALS clinical research.  Laura put this into practice at the ALS Association Advocacy Day in both 2011 and 2012.  Advocacy has become “a family thing”, something she and her family truly enjoy doing together and a great way to meet and interact with other PALS.  Her advice to PALS looking to be clinical trial advocates: “Don’t feel like you can’t do it.” She suggests have talking points prepared to make it easier to approach legislators about what the needs are for the ALS community and for ALS research. She has used this approach when she met directly with Congressman Barney Frank, (D-MA) to request his support for the MODDERN Cures Act.  The MODDERN Cures Act creates a new class of drugs called dormant therapies-medicines that may provide treatments for rare diseases such as ALS.  “As we were driving home from our meeting, I received an email from his legislative assistant that Congressman Frank had agreed to be a co-sponsor of the bill.  My family and I felt like we had made a difference by participating in advocating for ALS research.”

Where will she go from here? Laura says she will continue to do all she can to promote clinical trial participation for PALS. For PALS looking to get involved in ALS clinical trials, she encourages them to not only ask their neurologists about trials, but to explore the NEALS website and to keep options open and not limit themselves.  Talk to other PALS, call nearby universities and clinics and ask about opportunities, contact your local ALS Association chapter.  “It is amazing the courage and energy that you get when you’re facing a terminal illness.”

Thank you to Laura and all the other PALS who help us fight against ALS.

To learn more a
bout the importance of placebos in clinical trials, go here.  

There are currently over 100 NEALS clinics spanning the US, Canada, Mexico, and Ireland.  PALS and their families can use the NEALS website to assist them in locating an ALS specialist near them by going here.
 

MODDERN Cures Act: "The bill grants dormant therapies an additional period of regulatory protection, thereby encouraging more complex research and development initiatives, while also establishing a predictable timeline for the introduction of low-cost generics." (National Health Council, 2012).


The following comments have been received in response to this blog post. To leave your own comments, please click here.
Brainstorm Cell Therapeutics Stem Cell Therapy is the best. This therapy needs to be offered for ALS patients. The FDA needs to get out of the way for this treatment for ALS patients.
Comment left by Deniece Beckman on 08/18/2012
The current ongoing Brainstorm trial at the Hadassah Medical Center in Israel is investigating safety of mesenchymal stem cell implantation. Interim safety results indicated the NurOwn cell therapy was well-tolerated and did not present any undue risks. Efficacy of this treatment is not known. A clinical trial is planned for US in collaborations with researchers in Israel. We have had many inquires regarding the BrainStorm Stem Cell Therapeutics Clinical Trial. We will continue to follow the progress of the study in Israel and will keep our website current on all new trials and start dates (www.alsconsortium.org).
Comment left by ALS Trial Liaison on 08/20/2012
Deniece Beckman, my brother has ALS, first signs, May 2011, we have been reasearching stem cell options worldwide, have been considering NovaCells, but have unanswered questions still and reservations of course, iIam curious please as to your comment on Brainstorm Stem Cell and any info as to your conviction as to it being the "best" ? Thank you so much!!!
Comment left by Lester on 08/30/2012
Recent interim safety review on a phase 1 safety study indicated that transplantation was well-tolerated, appeared safe for use, and did not present any undue risks to the participants enrolled in the trial. This is a phase 1 safety study. Efficacy is not known. Therefore we can not assess whether it is the best therapy. There are many therapies under development for ALS. For more information about the science and technology behind NurOwn stem cell therapy, you can go to http://www.brainstorm-cell.com.
Comment left by ALS Trial Liaison on 09/07/2012
Presentation: Results of National ALS Registry Awareness Survey
Posted by: Rob Tison July 02, 2012
Rob Tisonhttps://docs.google.com/file/d/0B5nmEImgaRfudXc4ay1zUGNQa28/preview?pli=1
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Excellent work Rob! You are drawing attention where it is needed.
Comment left by ALS Trial Liaison on 07/03/2012
Has there been any action on the findings in Rob's survey? Thanks.
Comment left by C on 08/08/2012
Please be so kind to let me know, in which Stage is Ceftriaxone Trial? I am very interested, in learning about this issue, because my son has been diagnosed with ALS. Luz Maria August 20th 2012
Comment left by Luz Maria De Camarena on 08/22/2012
Hello C, I will look into this and get back to you as soon as I have information. Thank you for your question.
Comment left by ALS Trial Liaison on 08/22/2012
In July 2012, the DSMB for the NINDS-sponsored clinical trial of ceftriaxone (phase III) in ALS recommended that based on existing data the trial be stopped because the study was unlikely to reach the pre-determined efficacy criteria. The NINDS leadership concurred. Pre-clinical research identified ceftriaxone as a promising treatment for ALS therefore it was important for people with ALS to find out if the drug could be beneficial in ameliorating the disease. The study used a novel seamless adaptive design. Final analysis and presentation of the results will occur after completion of site monitoring and database lock. The important contributions of patients, their families and the hard work of the investigators and their teams made it possible to implement the trial. While all had hoped for a more positive result, the trial has moved ALS research forward. There are other ongoing trials investigating potential treatments for ALS. I would be happy to follow up with you to discuss clinical trials via your email, Luz
Comment left by ALS Trial Liaison on 08/22/2012
Open Access Is Not for Scientists. It’s for Patients.
Posted by: Rob Tison June 14, 2012
Rob Tisonhttp://blogs.plos.org/speakingofmedicine/2012/06/14/open-access-is-not-for-scientists-its-for-patients/
To leave a comment, please click here.
Acceptable Risk from a Patient Perspective: Neuralstem
Posted by: Rob Tison June 01, 2012
Rob TisonGreat TEDMED video with Drs. Glass and Boulis of Emory:

http://www.youtube.com/watch?v=-5QNrOJzvCs&sns=fb

Minor correction:. Not only 1 per 100,000 get the disease. It is 2 per 100,000 PER YEAR, AND REPEATS EVERY YEAR OF LIFE. So, it's more like 1 per 500 lifetime risk!
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Every Congressman in our country should see this post and video
Comment left by Deniece Beckman on 11/01/2012
Washington, DC Advocacy
Posted by: Rob Tison May 24, 2012
Rob Tison
Many travel to Washington, DC for advcocacy. So exactly what do we do?

The photo below is an example, with me talking directly to NC Senator Kay Hagan for a few minutes!

We meet in separate meetings at the offices of Senators and Congresspersons to share our "ALS Stories" and discuss our (the ALS Association's) public policy goals. This year they related to continued appropriations for ALS research and the National ALS Disease Registry, and a bill that could modernize policy regarding drug exclusivity, to add more incentives for unmet conditions.



My ALS Story:

I am Rob Tison, from Arden North Carolina, near Asheville, and a person living with ALS. I can no longer talk clearly and so have to use a computer to speak. I am a son, brother, husband and father of a 15 year old boy and 12 year old girl. I worked most of my career as a mechanical engineer. I have always been very athletic and also enjoyed the outdoors. In fact, I spent many years as a semi-professional road bicyclist while working as a full time engineer. I have also run multiple marathons and endurance trail runs. Other hobbies included whitewater kayaking, backpacking, hiking and mountain biking.

My life changed at the age of 39, on March 18, 2010, when I was diagnosed with Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease. ALS is a particularly cruel disease that destroys a person’s ability to control all voluntary muscle movement, often rapidly. As the disease progresses, I may become trapped inside my body; completely unable to walk, move my arms, breathe or even blink an eye.

There is no effective treatment for ALS, no known cause and no cure, yet the lifetime risk for everyone is about 1 in 500.

The disease also does not discriminate in who it strikes and can affect anyone at anytime, regardless of their age, gender, race or ethnicity. Military veterans are approximately twice as likely to die from ALS as those who have not served in the military, and it is now recognized as a service related disease.

ALS is always fatal – in an average of 3 years following diagnosis.

This disease quickly robbed me from my ability to speak. My hands were next and are very weak to the point where I can no longer do simple tasks like opening ziplock bags, lids on jars and bottles. Zipping my fly, doing buttons on shirts or tying my shoes are completely unachievable. I type with one able pinky finger. I can no longer walk even a few steps without support. My breathing is weakening too. Needless to say, I am no longer able to run, bicycle, kayak and backpack. I was not even self sufficient in slightly more than one year since diagnosis. I am now 100% reliant on others. My mental acuity is better than ever, so I am keenly aware of my condition and prognosis. I often think of ALS as the polar opposite of Alzheimer's Disease.

I am not here for myself, as it is likely too late for me. I am resolved to do what I can to ensure continued research and development of effective treatments for ALS, to benefit those more recently diagnosed or those in the future.

Thank you.



The following comments have been received in response to this blog post. To leave your own comments, please click here.
Rob, you are truly a gift to this community. Thank you, and your family, for your presence among us.
Comment left by Bob Murray on 05/26/2012
As always, Rob, your writing is so incisive and true that I have to thank you once again for sharing it with the rest of us. I was fortunate to go to DC this year for the first time and was very glad to see you there with your wonderful family. I was with the Massachusetts delegation, so visited different offices on Capitol Hill. It was worth the effort as among staffers we met, one did have a neighbor with ALS, one did not realize that Veterans are twice as likely to die of ALS, and several had questions about the progression of the disease and what our three requests would do. As you said we were asking for continued and increased support for the ALS Registry, DOD research projects and a new way to rescue previously patented drugs from the shelf so they can be tested in ways and on diseases different from the original testing. I told some of my story, too. I am 66 and a wife, mother and grandmother. I have bulbar onset ALS and my speech is gone and swallowing is extremely difficult now. I am lucky to still have the full use of my limbs and feel obliged to somehow use the strengths I retain to act for the people who die so quickly they never have a chance to work for a cure. I have donated blood, skin, and DNA to biomarker research and plan to have a PET scan of my brain in the next month so researchers can look for distinguishing 'signs' of ALS. Life is mysterious, and just as I do not wonder why me (why not me?), I do not wonder why I have the privelege of such a supportive family, such caring friends, and such amazing grandchildren. I can no longer read to my grandchildren or sing to them or praise their every move with words, but I can still hold them on my lap and they put up with my limitations. As one who is 3 1/2 said, some grandmothers can talk and some can't. But I do worry about how my passing will affect them. Like you, Rob, I think the work being done now deserves all the help we can give it, even though any new treatments or even cures will most likely come after we are gone. So we troop up to the offices of Congresspeople and raise awareness (and some money) by walking with our relatives and friends and carry on in support groups and on-line. You are an inspiration, Rob. Please know your work is helping us all. MM
Comment left by Mary Murray on 05/26/2012
Rob, thank you for all the work you do for all of us suffering with ALS. It was so uplifting to meet you and your wonderful family in Washington this year. I am very hopeful the cure is near!
Comment left by Susan Speranza on 05/29/2012
Congratulations!
Posted by: ALS Trial Liaison May 18, 2012
ALS Trial Liaison NEALS wants to extend our congratulations to blogger and advocate Rob Tison.  Rob won the ALS Association's Advocate of the Year Award at the ALS Association Advocacy Day this past week in Washington DC.  Each spring, thousands of ALS advocates gather in our nation’s Capitol in an effort to change laws and policies that affect PALS and their families.
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Add my congratulations to Rob! Well deserved! And thank you Rob for all the research and explaining you do here and on other forums like PLM. you help us all keep up to date and reasonably unconfused on important development in the world of ALS research! Thanks!
Comment left by Mary Murray on 05/18/2012
Thank you all! I was totally unaware of getting the award in advance. The award, but more so meeting people like Mary Murrary, Dr. Bedlack and the Tuttles again in DC rejuvenated my mental batteries!
Comment left by Rob Tison on 05/18/2012
Nobody has caused organizations or projects fighting ALS to move forward more than Rob Tison. He is smart, he is thoughtful, he challenges, he's a good egg.
Comment left by - on 05/18/2012
Congratulations Rob! I have enjoyed following your posts on ALS forums as you are continually positive and help others try to make sense of all the science out there. Keep on posting. . . you make a difference for all pALS!
Comment left by Laura Tuttle on 05/22/2012
Congratulations, Rob on being awarded the Advocate of the Year Award at the National ALS Association Advocacy Day and Pubic Policy Conference in Washington, D.C. It was a pleasure seeing you and your family again and I enjoyed standing with the rest of the audience paying tribute to you and the enormous effort you put forth for the ALS Community. Thank you for being a tremendous advocate.
Comment left by Tom Kettler on 06/01/2012
Please spread the word - National ALS Registry FEEDBACK Survey
Posted by: Rob Tison May 16, 2012
Rob TisonPlease help.  Late next month, I will be serving as a panelist with the National ALS Registry team with the ATSDR / CDC for the 3rd time, as a PALS representative.  I have been asked to give about a 15 minute presentation on PALS’ perception of the Registry and especially to discuss ways to reach out to all PALS. I have prepared a simple 10-question survey aimed at PALS' awareness of the Registry, on my own, located at the following weblink: www.surveymonkey.com/s/CYF8ZW2 I'd like to get as many responses as possible from PALS (or CALS on their behalf).  The target respondents are American PALS (or CALS). Please only one response per PALS (no need for PALS and CALS pairs to both respond.  Also, excluded are CALS of PALS who died before October 2010, since they could not register. Responses are 100% anonymous.  Please respond whether or not you have registered and whether or not you are satisfied with the Registry (to prevent selection bias). Thanks in advance!!! Rob
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Rob, please feel free to also post on the NEALS Facebook page. Link is https://www.facebook.com/NortheastALSConsortium. Thanks!
Comment left by ALS Trial Liaison on 05/18/2012
Rob, You are an inspiration to me. I, will do anything to help with research. I am 76 yrs old and want to see my great grandaughter grow up.
Comment left by Jim Fitzpatrick on 09/02/2012
Thanks to Legislators
Posted by: Bob Murray March 25, 2012
As we know, HR 2055, a comprehensive bill passed on December 17, 2011, included over  12,000,000 dollars for continued funding of ALS research related to veterans, and for the ALS National Registry for all PALS.  I did a little quick research and was happy to see that Mary's and my US rep, Steven Lynch, and US senators, John Kerry and Scott Brown, voted for the appropriation. We had spoken to Lynch at a local "Congress on your corner," and to Brown at an ALS walk in the fall.  [Not saying that tipped the scales :)] I called all three legislators this morning, talked to their aides, and asked them to insure that the principals were aware of our gratitude.  Just a drop,  of course, but we live in hope that lots of "drops" can add up!
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Those “drops” are just as much a part of ALS advocacy as all your other efforts. Well done.
Comment left by ALS Trial Liaison on 01/05/2012
Mary and I attended a St. Patrick's Day event on March 16, and Senator Scott Brown was present. We introduced ourselves, and he in fact remembered meeting Mary at the ALS MA walk last year. In fact, he related that he remembered her because at the time he did not realize that ALS could present with bulbar symptoms; and after meeting Mary, he was struck by the fact that she could not speak. He went on to say that he had attended a fund raiser earlier in the week with Ambassador Paul Celucci, who announced publicly that he too has ALS, and is attempting to raise million dollars for research. Sen. Brown assured us that ALS, and the need for continued funding, remains a top of mind issue for him.
Comment left by Bob Murray on 03/25/2012
Ambassador Cellucci's fund raising goal is 10 million dollars.
Comment left by Bob Murray on 03/25/2012
Patient Enthusiasm Toward ALS Clinical Trials
Posted by: Rob Tison March 09, 2012
Rob TisonWe often hear that clinical trial participation rates are low among PALS.  Could times be changing?

Just today, Biogen Idec's CEO, George Scangos, was quoted saying: "We had so many requests to get into ALS trial. By time closed, enrolled 942 when expected 800."  

This was in reference to the Dexpramipexole EMPOWER trial.  Enrollment closed in less than 6 months and was expected to take 10 months.  Many sites barely obtained IRB approval before enrollment closed, leaving active screenees turned away.

The NP001 phase 2 trial was similar. Due to PALS' enthusiasm and enrollment momentum, it enrolled 136 with a quota of 105 in about 7 months , 6 overlapping the EMPOWER trial.  Once again, the last few IRB's barely approved the trial before enrollment closed, and some with screening appointments were turned away. And, untold more willing PALS failed screenings, lived too far from clinics or knew they didn't meet eligibility. So, it seems there are unutilized willing and eligible PALS, due to a shortage of trial slots.

With this in mind, it is my sincere hope that phase 2 ALS trials will be increased in size to increase statistical power and enable more confident answers years earlier.  It's unacceptable to wait years in long phase 3 trials to finally learn of lack of efficacy.

Aim higher with less costly phase 2 trials to avoid risky futile phase 3 trials.  This may also allow for accelerated approval when an ALS drug shows effectiveness. Please don't design small trials because of pessimism regarding trial recruitment!
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Please read the timely related article: http://pipeline.corante.com/archives/2012/03/15/not_quite_so_accelerated_says_phrma.php Excerpt regarding the TREAT Act: "It would, ideally, encourage companies to run more robust Phase II trials in the hopes of going straight to the market, and it would allow really outstanding drugs a chance to start earning back their R&D costs much earlier"
Comment left by Rob Tison on 03/16/2012
Is there any possibility to enroll my husband in your trials? We live in Argentina and I´d had contact to Biogen Idec in Buenos Aires. Could you please inform about that? or where or to whon can I write to? Thank you very much! Best regards Florencia
Comment left by Florencia Souza on 05/23/2012
Dates and locations for the second Phase III trial of dexpramipexole have not yet been released, but as soon as they are announced, this information will be posted on the NEALS website. Contact information for each site will also be listed.
Comment left by ALS Trial Liaison on 05/29/2012
Like Us on Facebook!
Posted by: ALS Trial Liaison March 06, 2012
ALS Trial Liaison   NEALS is excited to announce that we are now on Facebook!  Be sure to log on and like us.  Here is the link:

http://www.facebook.com/NortheastALSConsortium
To leave a comment, please click here.
Webinar: Breaking Down Enrollment Barriers in ALS Research Through Advocacy
Posted by: ALS Trial Liaison February 16, 2012
ALS Trial Liaison
This week Richard Bedlack MD, PhD, spoke about strategies to overcome enrollment barriers in an ALS Association/NEALS webinar presentation titled, “Breaking Down Enrollment Barriers in ALS Research Through Advocacy.”  If you missed it, be sure to view it here.  

While there are many new exciting areas being delved into in ALS research, significant barriers, in particular low enrollment rates, still impede progress towards finding a cure.  In the most recent NEALS / ALS Association Webinar, Dr. Bedlack describes the important determinants of clinical trial enrollment.  He provides detailed examples of steps researchers are taking to not only promote enrollment, but to improve the design of clinical trials to make clinical research more appealing to patients.  Dr. Bedlack outlines “why”, “what”, and “how” PALS advocacy efforts can help foster new clinical research.  The webinar ended with several questions from the attendees.

The following comments have been received in response to this blog post. To leave your own comments, please click here.
i would like a list of all als drug trial clinics to post on a us map. are there are dead zones for coverage. i commute 190 miles one way to participate in my 2nd drug trial. could satalite clinics be established to reach more participants? i have asked for training to be an als ambassador to encourage more pals to sign up for trials. some trials languish for long periods of time due to no volunteers
Comment left by jerry fouts on 03/04/2012
Hi Jerry, Thank you for your comment. A map is something that we can look into doing for the NEALS site. At this time, you may want to use the Advanced Search Feature and run a search using your zip code. You can look for trials enrolling within 300 miles of the entered zip code. Satellite clinics are definitely a goal for researchers to ease participation of trials. This is where the CDC ALS Registry can become so crucial in helping to map out where PALS are in the country to know where to best begin to try to build more clinics. It is so wonderful to hear from PALS who are eager to participate in the Clinical Research Learning Institute! At this time, we are still determining a process for selection. As soon as we know, I will be in touch. Thanks again!
Comment left by ALS Trial Liaison on 03/05/2012
Hi Jerry, Clinicaltrials.gov has a map feature that shows state-by-state results. You can go our Navigating Clinical Trials.gov page on the NEALS website for instructions. Here is the link: http://www.alsconsortium.org/navigating_clinicaltrials.gov.php
Comment left by ALS Trial Liaison on 03/15/2012
ALS Association Efforts
Posted by: Tom Kettler February 15, 2012
I want to share some of the efforts I have been able to participate in through the National ALS Association. In mid January, the National ALS Association held the Annual Leadership Meetings in Nashville, TN. The purpose is to formulate strategic planning and policy designed to better the Associations ability to provide Care Services, Research funding and Public Policy. One of the events enjoyed by the entire group of participants was a Lunch to Honor our Heroes Living with ALS. One of the three honorees at that luncheon I first met at the NEALS CRLI Conference in October. She did a magnificent job of telling her story and finished to a standing ovation. Well done!

Another facet of the meetings was that the Chapter Relations Administrator invited chapter representatives to participate in a panel discussion entitled "Growing The Association's Research Program: How Can Chapters Make the Case?". A great amount of information was shared offering different approaches, philosophies and theories of how research is part of the "three legged stool" along with care services and advocacy needed to marry each Chapter's purpose and goals. I was able to share the CRLI experience in Clearwater Beach as an example of the need to advocate and fund research for our dedicated scientists and PALS. The panel generated a lot of positive questions and discussion as well as interest in the CRLI.  

The end of January was our National ALS Fly-In to Washington, D.C. where we were instructed on our Advocacy Priorities followed by visits to key legislative directors on the Hill. These meetings were preparatory to our visits to the Hill on National ALS Advocacy Day coming up in May. The three priorities presented to our legislators are:
(1) Appropriate 10 Million to continue the National ALS Registry At The Centers For Disease Control and Prevention;
(2) Appropriate 10 Million to continue the ALS Research Program (ALSRP) At the Department of Defense (DOD); and
(3) Enact the Moddern Cures Act, H.R. 3497.

For more information on each of these priorities please go here.  I will probably see some of you in Washington in May.



The following comments have been received in response to this blog post. To leave your own comments, please click here.
Thanks Tom! Keep us updated about your trip in May. It is also exciting to know that Clinical Research Learning Institute was so well received at these meetings. There seems to be a lot of interest out there in taking part.
Comment left by ALS Trial Liaison on 02/15/2012
It was great to see you in DC 2 weeks ago. Hope to see you in May.
Comment left by Rob Tison on 02/16/2012
Thank you very much for the updates. This site has bloomed into a great resource! I hope that in the next couple of months that we can learn some specifics of how the MODDERN Cures act would directly help those with ALS. When I try to read it http://thomas.loc.gov/cgi-bin/query/z?c112:H.R.3497: , I see a big focus on implementation of diagnostics, and that doesn't seem to me like a front-burner issue for the disease without good biomarkers. I understand how it could be attractive to the broader National Health Council membership, but I wonder if it's the most urgent legislation to chase specifically for ALS. Thanks for any insights.
Comment left by rkn on 02/17/2012
Thanks, Rob. See you in Washington in May. Just a quick reminder for everyone that Reps. Eliot Engel (D-NY) and Peter King (R-NY) are circulating a "Dear Colleague" Letter advocating the appropriation of Million for continuing support of the National ALS Registry. Please contact your representatives in Washington and ask them to sign on to the Letter. You can access the letter and your representatives by going to: http://capwiz.com/alsa/home/
Comment left by Tom Kettler on 03/06/2012
Exciting News Re. Another Phase 2 Trial for ALS
Posted by: Rob Tison February 14, 2012
Rob Tisonhttp://www.als.net/Media/5409/News/ The ALS Therapy Development Institute to Launch Phase II Clinical Trial of TDI 132 in ALS Patients
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See this also: http://online.wsj.com/article/SB10001424052970204062704577221220332840952.html?KEYWORDS=ALS
Comment left by Rob Tison on 02/14/2012
Thanks! Also, at http://www.alsconsortium.org/news_gilenya.php
Comment left by ALS Trial Liaison on 02/15/2012
Is there a good reason that NEALS is stressing safety and tolerability, but ignoring the efficacy aspect of this planned phase II trial? Quote: "This Phase II clinical trial will determine safety and tolerability of TDI 132 in patients with ALS.  It will yield important information on dosing and safety for a subsequent efficacy study.  NEALS is working with ALSTDI to design and conduct the clinical trial trial." Yet, we know that phase II trials are aimed at early efficacy review and continued safety. FDA Description of phase II: "Phase 2 includes the early controlled clinical studies conducted to obtain some preliminary data on the effectiveness of the drug for a particular indication or indications in patients with the disease or condition. This phase of testing also helps determine the common short-term side effects and risks associated with the drug. Phase 2 studies are typically well-controlled, closely monitored, and conducted in a relatively small number of patients, usually involving several hundred people." There are multiple instances of FDA approvals of drugs for life threatening confound based on phase II efficacy (e.g. HIV and cancer). ALS is certainly life threatening and in desperate need of an effective therapy. Urgency should cause ALS phase II trials to be designed for the same potential, IMHO.
Comment left by Rob Tison on 02/16/2012
ALS TDI / NEALS Gilenya webinar has been scheduled for March 12 and people may register here: http://www.als.net/ALS-Events/ALS-Webinars/Register/?wid=03122012
Comment left by Rob Tison on 02/16/2012
Thank you for your questions Rob. In drug development there are often several phase II type questions that need to be answered prior to embarking on larger phase III, long term efficacy studies. These phase II questions include safety, dose selection and assessment for preliminary efficacy. We are working closely with ALSTDI to develop the Phase II program for TDI 132. This Phase II program will assess safety, dosing, biological effect and preliminary efficacy. We recognize the urgency behind finding an effective therapy.
Comment left by ALS Trial Liaison on 02/17/2012
I see here, http://www.alzforum.org/new/detail.asp?id=3065, that two phase trials are being discussed.
Comment left by Rob Tison on 02/21/2012
CALBIO2012: Driven by Patients
Posted by: Rob Tison February 13, 2012
Rob TisonThis seems like a relevant conference for some of our ALS clinician researchers looking for ways to improve and accelerate clinical trials and/or share knowledge:

http://www.baybio.org/baybio-sites/calbio2012/program/

CALBIO2012 is a statewide conference produced by BayBio and BIOCOM that brings together the life sciences sector in California and beyond: company executives and innovators, government, medical research and disease foundations, university transfer offices, philanthropists and investors.

Built on the synergy between two leading life sciences clusters, CALBIO2012 identifies best practices and new models and fosters collaborations in research, development and product commercialization.


Especially these tracks & sessions:

http://www.baybio.org/baybio-sites/calbio2012/tracks/clinical-development/
Track 2
Clinical Development
Thursday, March 8, 2012

Session 1
Patient Advocacy: The Key to the Next Evolution of Clinical Development

The sleeping giant in clinical development is the patient, both as the crucial resource in clinical trials and as a previously under-utilized source of clinical/molecular data and biospecimens. Increasingly, product innovators are viewing patients as full partners. Novel collaborations with patient advocacy groups – who have access to large numbers of patients with a particular disease, who are motivated to drive and accelerate research, and who can bridge the silos of academe and industry – are emerging. Patient groups can also be supportive to the development of new models, such as adaptive clinical trials, that have the potential to produce better clinical outcomes in a shorter time frame. This session will explore the pro-active role of advocacy groups, how these types of collaborations are taking shape, how they might fuel a new generation of clinical development, what is needed to harness the potential of all the data, and how to avoid some of the common pitfalls.

Session 2
Ten Tips to Accelerate Clinical Trials and Do Them Well

In order to expedite the speed with which clinical trials are completed, it is critical to have a solid foundation for measuring and tracking where key improvements in process and efficiency can be made. There are general areas where sponsors can improve the speed of clinical trial completion, for example: decreasing time for subject enrollment and recruitment, decreasing site contracting time and speeding up site initiations. In this cost constrained environment, however, there is a cost benefit sponsors are willing to accept in order to save time and improve clinical trial performance. Due to the cost of making improvements, it is important to have key processes in place for identifying where changes can have the biggest bang for the buck. Processes for identifying key changes include: -Developing a balanced scorecard for selecting the right sites and investigators -Key criteria for streamlining the feasibility process -Establishing enrollment forecasting methodology -Optimal incorporation of feasibility and subject enrollment Once processes such as these are established, key areas can be identified for improvement. This approach has the benefit of decreasing sponsor expense by honing in on specific areas of improvement while accelerating trial performance and efficiency.

Session 4
Modernizing our Industry: Developing the Clinical Trials of the Future

The average drug costs over a billion dollars to develop, and takes more than ten years to get from the bench to the patient. Without question, the costliest, most time-consuming part of drug development is the process of clinical testing, however, most clinical trials are conducted in much the same way that they have been for the last 50 years. Could the cost of conducting clinical trials be reduced? Could we speed up the time in which it takes to get a drug through clinical testing? And, is it possible to design better clinical trials so as to avoid costly late-stage failures? New technologies and approaches now make the answers to all of these questions a resounding “yes”. This panel will focus on the future of clinical trials and what is being done to modernize the process of drug development. Panelists will discuss new recruiting techniques, mobile health technologies for remote patient monitoring, innovative trial designs and industry-wide consortiums that will ultimately revolutionize the way we conduct clinical trials, reducing costs and speeding drug development.
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http://www.baybio.org/baybio-sites/calbio2012/tracks/patients/
Patients
Track 3
Friday, March 9, 2012

Session 1
Taking Matters into Their Own Hands: The Role of Patients in Drug Development 

In major world markets, patients have emerged as a significant power in drug development, approval, reimbursement and communication. Patient organizations are playing a critical role in many cases, from co-creation of innovative therapies and delivery systems, to faster approval and wider reimbursement. For biopharma companies, a successful business model now relies on involving patients and advocacy groups in each stage of development: joint drug development and testing (injectable biologics such as Novo Nordisk’s insulin reached global success due in part to patient-friendly pens); collaboration in speeding approval from regulators, such as the role of patient groups in the fast approval of Novartis’s leukemia drug, Gleevec; understanding of patients’ experience of disease, such as Sanofi Genzyme’s close links with families of patients with Gaucher disease and other orphan diseases. This panel representing innovating biopharma companies, leading foundations/advocacy groups and key industry experts will explore the role that disease foundations and patient advocacy groups can and should play and discuss current best practices in successful company-patient collaborations throughout the value chain.

Session 2
Patient Access to Developmental Drugs

This session explores proposed regulatory innovation in the area of life-threatening diseases that have unmet medical need. Since the 1962 tightening of evidentiary standards for safety and efficacy, FDA has struggled to balance benefit and risk in a way that helps patients in need of treatment. Our one size fits all approach to drug approval has not allowed individuals to express their own preferences for risk versus potential health improvement, especially in deadly diseases for which no approved treatments are effective and the only chance at survival may lie with new investigational drugs. The panel session considers the effectiveness of FDAMA’s Accelerated Approval provisions and whether they have produced a net benefit to terminally-ill patients over the last 15 years. In the last 10 years, several initiatives for more meaningful progressive regulatory approval have been argued in Congress and in the Judiciary, including parallel-track and class-level authorization. We will examine these initiatives and discuss new draft legislation proposed by BIO and by Senator Kay Hagen (D-NC). The implications are not just human lives, but also dramatic reduction in regulatory risk for companies considering development in deadly unsolved diseases.

Session 3
Social Media and Life Sciences: Hearing a Patient’s Voice

700 million of anything is hard to ignore. The exponential growth of social media, with nearly 700 million Facebook users, is well documented with increasing numbers of life sciences companies looking at ways to interact with healthcare providers, patients and consumers through social media. The key question, however, is how do you connect the right people with the right message at the right time? During this panel discussion you will hear about latest social media developments and trends, limitations and major pitfalls. Learn about key innovators and what experiences might be relevant for your company.

To leave a comment, please click here.
National ALS Registry
Posted by: ALS Trial Liaison February 09, 2012
ALS Trial Liaison

I want to share an example of what PALS are doing to advocate for ALS research.  

This YouTube video promotes the National ALS Registry, supported by the Centers for Disease Control.  The ALS registry can play a vital role in learning more about ALS, such as risk factors for the disease or patterns over time.  The more PALS who enroll, the more valuable the information derived from it.  Registration is easy and gives access to surveys, which family members and caregivers are encouraged to get involved with.  By registering, you will be contributing to ALS research and potentially helping PALS in the future. 

Listen to Rick as he talks about why the registry is so important and about benefits for PALS.  Thank you Rick!

YouTube ALS Video

CDC Registry

 

To leave a comment, please click here.
ALS Biomarker Efforts
Posted by: Bob Murray January 30, 2012
We all know how important biomarkers are to research generally, and how helpful it could be for ALS research in particular if there were reliable signposts for clinicians, scientists and and pharmas to use in their work. There are secure repositories at many ALS clinics that store blood and other types of samples donated by both PALS and non-PALS folks (the non-PALS for controls).  So we thought we'd do our bit at MGH. I've donated skin and blood, and have signed up for spinal fluid.  As a PALS, Mary has donated skin and blood.  The more folks who choose to support such work, the better the chance to discover some reliable biomarkers for ALS.
Bob Murray (may I respectfully add Go Patriots!)
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Biorepositories and biomarker trials are of the utmost importance. These samples can be used to speed diagnosis and begin to learn about the causes of ALS. Any PALS and non-PALS interested in giving samples should contact their local ALS clinic to see about opportunities, or take a look at recruiting observational studies in the NEALS database. You can also contact the ALS trial liaison at (877) 458-0631. Thank you for sharing Bob!
Comment left by ALS Trial Liaison on 02/01/2012
In addition to blood and spinal fluid type donation opportunities, which I ALS find incredibly valuable, I've recently realized there is no summary to be found for institutions accepting brain and spinal cord donations upon death for ALS research. I feel there is an unmet need there, as more people would participate if knowledge was more prevalent. These do not appear at clinicaltrial.gov or the new NEALS study search tools. Sorry for a morbid topic, but a summary of opportunities is needed for PALS.
Comment left by Rob Tison on 02/04/2012
Thanks Rob, you bring up a very good point about the need for this information. It is clearly a very sensitive topic, but does need consideration. There is a shortage of brain and spinal cord tissue for ALS research; a reason for this MAY have to do with lack of knowledge. There is now a list on the NEALS website of facilities that accept postmortem tissue donation. Thanks to the ALS Association, you can find this list at http://www.alsconsortium.org/trials.php. It is reviewed and updated as needed to ensure that it remains current. I will be working to get this information out in other formats as well. Thank you for bringing this up.
Comment left by ALS Trial Liaison on 02/07/2012
The ALS Research Pipeline
Posted by: ALS Trial Liaison January 18, 2012
ALS Trial Liaison
As Dr. Merit Cudkowicz stated in a recent lecture to colleagues, patients, and caregivers, “the pace of (ALS) discovery is increasing.”  While Riluzole is currently the only approved drug for ALS, many other treatments are being investigated for their potential roles in ALS treatment.  Researchers are improving upon clinical trial design and dosage; they are learning from past trials and using this information to design trials that are faster, more precise, and more appealing to participants.  What is coming down the ALS research pipeline?

Interventional Drug Trials
Cytokinetics has two actively enrolling trials continuing to investigate CK-2017357, a drug that enhances muscle contraction, one for two weeks and another for four weeks with the goal of arriving at an appropriate dosing schedule to use in a planned Phase 3 trial:

Enrolling Study #1
The phase II, randomized, double-blind, placebo-controlled trial is seeking to change the previous dose by including low, mid, and high dosage both without (Cohort A) and with (Cohort B) concurrent use of Riluzole.  Results from Cohort A show that CK-2017356 was well-tolerated when dosed daily in ALS patients.  Cohort B is currently enrolling; patients will reduce their Riluzole dosage to 50 mg for seven days before taking daily oral doses of either placebo, 125 mg, 250 mg, or 375 mg of CK-2017357 for fourteen days, and to remain at that dose of Riluzole while randomized.  
Enrolling Study #2
Cytokinetic’s titration study is a three week dose escalation study to see if subjects can tolerate 500 mg CK-2017357 in daily divided doses. Participants reduce their use of Riluzole to 50 mg for seven days before being randomized to receive twice daily doses of CK-2017357, or placebo, for seven days, totaling 250 mg, 375 mg, and 500 mg daily.  Researchers are examining the optimal dosage to display maximum tolerability and clinical effects.

Nuedexta is a drug approved by the FDA for improving emotional lability in ALS and other disorders.  Based on patient experience, a new NEALS trial will be starting soon to evaluate whether this drug improves bulbar functions such as swallowing and speaking.  Information regarding inclusion criteria and study sites will be posted soon.

The University of Kansas is investigating Rasagiline in a phase II, open-label trial to determine if it is safe in ALS patients and if it has the potential to slow ALS disease progression.  Rasagiline has possible neuroprotective properties.  Participants are being enrolled at 3 US sites: California Pacific Medical Center in San Francisco, CA, the University of Pennsylvania, and at the coordinating center, the University of Kansas.

Stem Cell Trials
Sponsored by NeuralStem, 12 participants received injections of human spinal cord-derived neural stem cells in the lumbar spinal cord at Emory University in Atlanta, GA. The next participants will receive injections into the cervical (neck region) spinal cord. This phase I trial is primarily designed to investigate safety in patients with ALS. Participants must live in close proximity to Atlanta.

The Northeast ALS consortium is working with Brainstorm Cell Therapeutics to design a Phase II clinical trial in the United States.  Brainstorm Cell Therapeutics is performing a Phase I/II clinical trial at the Hadassah Medical Organization in Israel, and is currently enrolling ALS participants in a trial of autologous transplantation of Mesenchymal Stromal Stem cells secreting Neurotrophic factors (MSC-NTF), taken from the patient’s own bone marrow.  The cells are delivered into the muscles or into the spinal fluid.
 
Other Interventional Trials
Phoenix Neurological Associates is investigating Zinc in a phase I/II, open-label trial to determine tolerability and safety of zinc at high doses in conjunction with copper, used concurrently with Riluzole for treating ALS.  Zinc may play a role in pathological processes associated with ALS.

Research is not only delving into drug treatment, but symptomatic treatment through exercise.  Until now, a randomized, controlled, large study evaluating the potential benefits of resistance and endurance exercise in ALS has not been systematically undertaken.  The purpose of this ALSA-funded trial titled “Trial of Resistance and Endurance Exercise in Amyotrophic Lateral Sclerosis” is to evaluate the safety and tolerability of resistance or endurance exercise in research participants with ALS. In this pilot study, 60 participants with ALS will be enrolled at 4 clinical centers.  Each subject will be assigned to treatment in 1 of 3 arms:  resistance exercise, endurance exercise, stretching and range of motion exercise.  Each participant will complete 6 months of this exercise regimen. Primary outcome will be tolerability of exercise regimen.

Enrollment Closures
Ceftriaxone, a phase III, randomized, double-blind, placebo-controlled trial investigating efficacy and long-term safety of Ceftriaxone, has closed enrollment.  Five hundred and thirteen participants were enrolled at 62 centers across the US and Canada. The study will close when the last enrolled participant has been in the trial for one year.  Ceftriaxone is an intravenous antibiotic that has several neuroprotective properties.  

The NP001 trial, a randomized, double-blind, placebo-controlled Phase II trial examining the effects of NP001 administered intravenously on clinical function at two dose levels, has also concluded enrollment.  The trial enrolled 136 patients at multiple study centers.  NP001 is a novel regulator of macrophage activation and a regulator of neuroinflammation.

Study Closures
In Cytokinetics’ completed phase II trial “A Study of CK-2017357 in Patients with ALS,” the effect of a single dose of CK-2017357 was measured in terms of safety and skeletal muscle function or fatigability.  Results were promising, in that a single dose of study medication reduced measures of muscle fatigue and was associated with perceived improvement by patients.  
CK-2017357 is now being investigated for optimal dosage in two other phase II trials.

NEALS is in discussions with several companies and foundations about potential therapies and will post study specific criteria and results of trials as soon as they are available to us.  NEALS promises to keep the ALS community at the forefront through its:

•Newly launched NEALS website, which contains a searchable ALS trial database that is monitored and kept up-to-date at all times.
•Clinical trial news and findings posted on the NEALS Clinical Trial News page.
•Introduction of a clinical trials expert, who is available for questions and response in a timely manner.



The following comments have been received in response to this blog post. To leave your own comments, please click here.
Thanks for the summary. Are you able to provide a forecasted start date for the second phase III trial of Dexpramipexole (Endeavor)? How about confirming another phase II trial of NP001 soon?
Comment left by Rob Tison on 01/12/2012
Thanks Rob, Information has not yet been released for either of those trials. As soon as it is, I will post it in the NEALS news.
Comment left by ALS Trial Liaison on 01/12/2012
you could give us further information about new phases or possible dates for the second phase III trial of dexpramipexole? Thanks.
Comment left by koke on 01/17/2012
At this time, nothing has been released about dates for the second phase III dexpramipexole (Endeavor) trial. When this, and other trial information, is announced, it will be posted on the NEALS website.
Comment left by ALS Trial Liaison on 01/17/2012
In reference to the Brainstorm information shared earlier in this blog, I read Brainstorm's January 17, 2012 comments on the status of their adult stem cell ALS safety trial. I found it interesting and potentially promising, albeit very early days, and the evidence is based on the study of only 4 ALS patients to date. Here is Brainstorm's announcement, January 17, 2012 BrainStorm Announces Clinical Data Supporting Safety and Efficacy of NurOwn™ Based on Initial Patient Results Business Wire | 1/17/2012 BrainStorm Cell Therapeutics Inc. BCLI, an innovative developer of adult stem cell technologies and Central Nervous System (CNS +1.31%, news) therapeutics, today announced that the data from the initial patients in its ALS Phase I/II human clinical trial treated with its NurOwn™ technology did not present any significant side effects and that the NurOwn™ treatment has so far proven to be safe. Prof. Dimitrios Karussis, who is leading the clinical trial at Hadassah Medical Center, stated, “There have been no significant side effects in the initial patients we have treated with BrainStorm’s NurOwn™ technology. In addition, even though we are conducting a safety trial, the early clinical follow up of the patients treated with the stem cells shows indications of beneficial clinical effects, such as an improvement in breathing and swallowing ability as well as in muscular power. I am very excited about the safety results, as well as these indications of efficacy, we are seeing. This may represent the biggest hope in this field of degenerative diseases, like ALS.” After reviewing the safety data from the first four patients, the Hadassah Medical Center ethical committee granted approval for the trial to advance to transplanting the next patients. “We are happy to report that the first patients treated with our NurOwn™ technology did not present any significant side effects. This supports and strengthens our belief and trust in our technology. Based on the interim safety report, the hospital ethical and safety committee granted the company approval to proceed with treating the next patients. We are pleased with the progress we are making and look forward to continuing to demonstrate the safety of NurOwn™ in the future,” said Chaim Lebovits, BrainStorm’s President. The ALS Phase I/II human clinical trial is being performed at Hadassah Medical Center in Israel in collaboration with BrainStorm and is utilizing BrainStorm’s NurOwn™ technology for growing and modifying autologous adult human stem cells to treat ALS, often referred to as Lou Gehrig's Disease. The study is headed by Prof. Karussis, M.D., Ph.D., who is the head of Hadassah's Multiple Sclerosis Center and a member of the International Steering Committees for Bone Marrow and Mesenchymal Stem Cells Transplantation in Multiple Sclerosis (MS +5.97%, news), and a scientific team from BrainStorm headed by Prof. Eldad Melamed. The initial phase of the study is designed to establish the safety of NurOwn™ and will later be expanded to assess efficacy.
Comment left by Mary Murray on 01/19/2012
Searching for Clinical Trials - Great New Tools!
Posted by: Rob Tison December 24, 2011
Rob TisonI am honored to been invited to be an ALS clinical research ambassador.  Even beforehand, I have realized the great importance of clinical trial participation to advance potential therapies, and even knowledge if trials should be unsuccessful.

Slow enrollment delays progress towards important answers, costs trial sponsors more money and may effect future trial sizes as sponsors size trials based on timing goals and historic participation rates.  Smaller trials may reject beneficial drugs due to lack of statistical significance. And, there are likely more drug candidates than the eligible, aware and willing patient population can support by filling trials.

Having been an active participant in multiple ALS forums for about 2 years, I've realized most PALS are very unaware of all recruiting trials, and this is a major factor regarding participation, among the willing and eligible.

The recently launched NEALS advanced trial search tool is a huge advancement.  It is user-friendly and powerful, with disease specific filters (e.g. fALS vs. sALS, no placebos, stem cells only, only active and recruiting, time since onset limit, FVC limit, etc.).  The www.clinicaltrials.gov site was the 'gold standard'.  It is complete, but is NOT user friendly and returned a daunting number of trials to sort through, making it impractical unless one wanted details about a trial they were already aware of and searched by keywords.  The new tool has the power of complete data, but allows 'intelligent' and practical filtering impossible elsewhere.  It empowers PALS!

The link to the advanced trial search tool is:  http://www.alsconsortium.org/search.php?v=a

Check it out and spread the word!  Increased trial awareness will result in increased participation.  Increased participation will lead to effective therapies sooner.

If you have a specific trial in mind, the basic trial search tool is also useful, using keywords: http://www.alsconsortium.org/search.php

And, if one is not confident in their search abilties or simply wants to interact with an expert, another terrific option is calling or emailing a NEALS Clinical Trial Expert with your questions about clinical research at (877) 458-0631 or alstrials@partners.org
rial Expert
The following comments have been received in response to this blog post. To leave your own comments, please click here.
Rob, thank you for your post. Please be sure to let other participants in your forums know as well. You are right; clinicaltrials.gov has a wealth of information, but can be overwhelming, especially if you are looking to minimize your results by looking for certain criteria. We truly hope that visitors to the NEALS site will use the trial search function and will find it to be a helpful tool. If anyone needs additional assistance, I am available over the phone to walk people through as they use the search tool. Any feedback about this feature or misinformation can be directed to me so that I may better assist you. Any trial question at all, please call me!
Comment left by ALS Trial Liaison on 12/27/2011
I agree with Rob that the NEALS site is really a big step forward in making the trials clearer for potential participants. I have been participating in the Empower trial by Biogen Idec of dexpramipexole since April and I thought it might be of some interest to someone considering participation If I told what it has been like for me. Far from feeling like an experimental object, I have felt welcomed as a co-participant in an important research project that may help many future ALS patients. I take a pill morning and evening(with applesauce since I have Bulbar onset ALS and have trouble swallowing) that may be either 150 mg. of depramipexole or a placebo. The trial requires monthly meetings with the team for checkups and blood draws (painless in the hands of experienced clinicians) and every three months, a more extensive physical by a doctor to determine how I am doing overall. Regularly, I have the FRS(questions that are used to scale ALS progression of symptoms) and the breathing test familiar to PALS. Some blood draws had to be taken at specific times after or before the pill was taken, but the team is very helpful in scheduling these to be as little of a burden as possible. Overall, the experience has been very positive for me. I may be on a placebo or the real 'dex' tablet, but time will tell. Either way, I feel I am contributing to the body of knowledge about ALS and its thus far unpredictable progression. Anyone considering participation in a trial should read all the material available on the trial and understand the risks and benefits. We need to find new treatments for ALS and we can only do that if we work together with those willing to do the hard science.
Comment left by Mary Murray on 01/04/2012
Mary, what you’ve said is so important. For many PALS, their lack of awareness may be what is stopping them from enrolling in trials. For others, just having the information may not be enough. Hearing and internalizing clinical trial experiences of other PALS can be a catalyst for change. That being said, you are right about reading all material first, because one trial might not be right for everyone. Thank you! I ask others who have been enrolled in trials, what positive experiences do you have to share?
Comment left by ALS Trial Liaison on 01/05/2012
Trials are terribly important, equally important is allowing early access to treatments that are in trial and demonstrating safe dramatic improvement. PALS are dying each day and early access is the humane step to take. 1800 of us have signed this petition. To learn more email: treat.us.now@gmail.com, or edjohnson@alumni.duke.eduem http://www.change.org/petitions/corporate-citizens-authorize-and-make-available-compassionate-use-drugs-for-als-patients-now
Comment left by ed johnson on 01/10/2012
Emergency Data Base
Posted by: Thurza and James Campbell December 15, 2011
I'm Chair of our town's disAbility Advisory Committee. We worked with Police and Fire on establishing and maintaining a confidential data base of all residents with any special needs. In power outages, they contact anyone on the list, giving the power company a heads up too, regarding those on oxygen, a bipap or respirator. If needed they deliver and hook up a generator or take the resident to a shelter. Every city and town needs such a data base. Police worked with the Council on Aging to compile the lists then Police visited each person to help them fill out the forms. We used schools to determine kids.  we publicized the project to the entire community, as all ALSers and others with special needs aren't seniors. Of course pride and HIPPA prevent any data base  being 100% complete.
i have the forms, but don't know how to attach them.
Thurza Campbell, PLS
Sherborn, MA
The following comments have been received in response to this blog post. To leave your own comments, please click here.
GOOD WORK THURZA!
Comment left by Marilyn and Larry Sanford on 12/14/2011
Thanks Thurza! It is important to remember that advocacy can go beyond research to include the crucial issue of safety such as this.
Comment left by ALS Trial Liaison on 12/16/2011
Thanks Thurza! On a related note, it occurred to me the other day that my wife, Mary, with advanced bulbar ALS, can no longer speak - and how would she reach E911 for an emergency (and not limited to ALS related of course). I've started some research, early days, and a TTY device seems one reliable way forward (for a PALs who can type). It's essentially a keyboard that allows messages to be typed over an ordinary phone line, and E911 in Massachusetts has TTY devices that allow the E911 agent to read the "caller's" emergency description. The devices can cost 300 or more from what I see on line - insurance, Medicare, Medicaid, etc may cover some of the cost as a piece of durable medical equipment. And the need applies not only to the PALS own emergency (if the CALS is not at hand e.g.), but suppose a CALS needs an E911 response, and the PALS cannot speak to get help. As I say, early days. One final thought - if a PALS can dial E911, the agent is expected to dispatch a responder if the line remains connected though no one speaks. But it might be a police officer rather than a medical unit.
Comment left by Bob Murray on 01/04/2012
More info - I realize this blog site is essentially regarding clinical research, but along Thurza's line, safety is important of course, and I wanted to add what I've just learned regarding E911 in MASSACHUSETTS - helpful for non speaking PALS who can type. The state's Office of Public Safety outlines what's called MASSACHUSETTS SILENT CALL PROCEDURE: Dial 911, wait for agent to answer, then press 1 for Police, or 2 for Fire, or 3 for Ambulance from a touch tone land line. See http://www.mass.gov/eopss/agencies/state-911/massachusetts-silent-call-procedure.html In addition, the MASSACHUSETTS E911 staff can source assistance devices (TTY and others) for "disabled" folks who need them to reach E911; an income means test determines how much folks might pay. See MassEDP http://www.mass.gov/eopss/agencies/massedp/
Comment left by Bob Murray on 01/04/2012
Use What You Got
Posted by: Richard Bedlack, MD, PhD November 29, 2011
Richard Bedlack, MD, PhD
People think I’m nuts.  They say that doctors aren’t supposed to have hair that looks like they just rolled out of bed, or loud clothes, or tattoos, or a purple car.  But this is who I am. Thankfully, it turns out that these eccentricities can be effective weapons in my fight against ALS as well.  

For example, I am sometimes asked if my hair is for real, and how it stays that way.  I tell people it is from a combination of cheap mousse and great surprise…the latter due to the fact that less than 10% of patients with ALS ever enroll in research studies!  To me this means that our studies cost more, take longer to complete, may end without an answer and may not be generalizable to all patients with ALS…in short that it will take longer to find the cure for this terrible disease.   This would make anybody’s hair stand on end!

I am sometimes asked why I wear such brightly colored suits.  Well, these reflect my optimism:  I know we can fix this enrollment problem. NEALS research that shows that the major determinants of enrollment are doctor factors and patient factors…things like lack of awareness of studies, time and money burdens, and surprising misconceptions about research in general and about alternative and off label therapeutic options.  Initiatives are underway to address these issues, including a clinical trials concierge service, expanding the numbers of sites that offer trials, educational materials such as webinars and video-assisted consent, studies that have home-based outcome measures, and ALSUntangled, (alsuntangled.org) which helps patients and their families use social networking to investigate alternative and off label treatment options.

Most often I am asked about my swallow tattoo; this opens the door for me to talk about Tim.  When this talented videographer and musician’s mom died from ALS, she left him a book full of hand-drawn swallows; later he had these tattooed onto his arm.  When Tim got the disease his family and friends got their own swallow tattoos, and came together to create the Often Awesome Army (oftenawesome.org).  Turns out the swallow is a very old tattoo that sailors used to get; it was the first bird they saw when land was approaching and thus let them know they were not lost at sea.  What a perfect symbol for all of us fighting ALS!  Over the past 2 years, Tim’s Often Awesome Army held numerous highly publicized and well-attended concerts, art shows and tattoo marathons, and they created a series of You Tube Videos that have been watched more than 10,000 people.  Tim showed me the power individuals have when they direct their energy and unique talents toward their fight.   To similarly empower others, NEALS recently held the first ALS Clinical Research Learning Institute.  We helped educate a small group of incredibly talented patients and families about ALS research and advocacy; we look forward to seeing how these new “research ambassadors” meld their unique skills and newfound knowledge to help us improve enrollment in ALS research.

Readers, I am sure you have your own unique talents.  What makes you special or different?  Can you think of a way to use what you got to educate and advocate for some aspect of ALS research?  Remember the words of Margaret Meade: “Never doubt that a small group of thoughtful committed citizens can work together to make a difference.  Indeed that is the only thing that ever does.”  Celebrate your eccentricities, make them work for your own fight against ALS…these individual efforts are not only fun, but when added together they will sum to a powerful force.

Oh yeah…and my purple car?  Well if that turns your head, you can’t miss my license plate:



The following comments have been received in response to this blog post. To leave your own comments, please click here.
As simple as it sounds, talking and listening go a long way. Patients who enroll in clinical trials want to know the value of their contribution. Perhaps if we increase communication with patients not only before and during, but after trials, participants can help shape understanding of ALS trials to other patients. You asked what I got…well, I’ve got the concerns and questions of pALS and caregivers. I’ve got clear, relevant, accurate information to give back to them. I hope to make navigating the ALS clinical trial world a lot less daunting. And a big thank you to our new research ambassadors. Without you, the field of ALS advocacy would not be moving at the rate it is. Keep up your amazing work!
Comment left by ALS Trial Liaison on 12/13/2011
Enjoyed meeting you in Clearwater. LOVE the license plate!!
Comment left by Marilyn & Larry Sanford on 12/14/2011
Welcome
Posted by: ALS Trial Liaison November 28, 2011
ALS Trial Liaison
Welcome to the NEALS Clinical Research Blog!  One of the new features on the NEALS website, the blog is an interactive tool written by ALS Association / NEALS representatives and research advocates to report on clinical research advocacy efforts and other clinical trial information.
The following comments have been received in response to this blog post. To leave your own comments, please click here.
As of 1/1/12 there are over 30 new Treatments for ALS (not counting Stem Cell treatment) undergoing FDA trial. We are dedicated to having some of these made available as soon as they meet the FDA minimum requirement for Compassionate use..... The drug companies and the FDA are being asked to Treat Us Now! As soon as efficacy becomes apparent during Phase II begin taking the necessary manufacturing steps to have available supply for those PALS willing to sign off on accepting potential risk. Assure that Drug companies understand the Public Relations benefit from supporting early access. PALS will have to arrange funding and monitoring for access to early treatment. http://www.change.org/petitions/corporate-citizens-authorize-and-make-available-compassionate-use-drugs-for-als-patients-now email:treat.us.now@gmail.com, or edjohnson@alumni.duke.edu
Comment left by ed johnson on 01/10/2012
 

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