Mission Statement

The mission of the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is to translate research advances rapidly into clinical trials for patients with ALS.

Accomplishments

In 1995, investigators from nine northeast region academic institutions, sharing a special interest in ALS, came together to form the Northeast ALS Consortium. They created the Consortium to solidify research that identifies drugs with therapeutic potential, and to send those drugs swiftly into clinical trials for patients with ALS.

Since then, the NEALS Consortium has welcomed investigators from throughout the North America, and now comprises 70 ALS clinics and research centers from coast to coast. The Northeast ALS Consortium has become a leader in ALS clinical trials.

Since 1999, the NEALS Consortium has initiated and completed three investigator-led clinical trials in ALS. The NEALS Consortium initiated the first clinical trial in ALS to be funded by the National Institute of Neurological Disorders and Stroke (part of the National Institutes of Health). The purpose of the trial was to study the efficacy of topiramate in slowing disease progression in subjects with ALS. Two hundred ninety-six patients from 20 sites enrolled, and the results were published in Neurology. (2003; 61:456-464.)

The NEALS Consortium also completed a trial of creatine in ALS (Neurology 2004;63:1656-1661, Muscle and Nerve 2004; 30:463-469), and a trial of Celebrex in ALS.

The Northeast ALS Consortium was the first group to use Motor Unit Number Estimation as an outcome measure in a clinical trial. NEALS has established criteria for the reliability of other disease outcome measurements, including Quantitative Muscle Testing (QMT), Forced Vital Capacity (FVC), Maximum Voluntary Isometric Contraction (MVIC), and the ALS Functional Rating Scale (ALSFRS.) Currently NEALS is pioneering the development of hand held dynamometry (HHD) as an outcome measure, and is testing an ALS-specific quality of life instrument.

The NEALS Consortium established its own Clinical Coordination, Project and Data Management Center, located in the Neurology Clinical Trials Unit at the Massachusetts General Hospital, in Boston.

The NEALS Consortium brought together leading ALS scientists with ALS clinicians and clinical researchers. A Scientific Advisory Board was formed to offer advice on the best therapy selection and direction of research.

 

Leadership

Chairs

Merit Cudkowicz, MD, MSc
Jeremy Shefner, MD, PhD

Executive Committee

Robert H. Brown, MD, DPhil
Lora Clawson, MSN, CRNP
Merit Cudkowicz, MD, MSc
Julaine Florence, PT, DPT
Terry Heiman-Patterson, MD
Jeffrey Rothstein, MD, PhD
David Schoenfeld, PhD
Jeremy Shefner, MD, PhD
Rup Tandan, MD, FRCP
NEALS Program Manager (non-voting member)

Scientific Advisory Board

M. Flint Beal, MD
Robert H. Brown, MD, DPhil
Don Cleveland, PhD
Robert Ferrante, MS, PhD
Robert Friedlander, MD, PhD
Jeffrey Rothstein, MD, PhD

 

Profiles

Robert H. Brown, Jr., MD, DPhil

Dr. Robert H. Brown is Director of the Day Neuromuscular Research Laboratory and the Muscular Dystrophy Association (MDA) clinic at the Massachusetts General Hospital. He is a Professor of Neurology at the Harvard Medical School. Dr. Brown graduated from Harvard Medical School and completed his doctoral training in Neurophysiology at Oxford University. Dr. Brown trained in Neurology at Massachusetts General Hospital.

In 1984, Dr. Brown founded the Day Neuromuscular Research Laboratory to investigate neuromuscular diseases, including Miyoshi myopathy and ALS.

 

Lora Clawson, MSN, CRNP

 

Merit Cudkowicz, MD, MSc

Dr. Merit Cudkowicz is an Associate Professor of Neurology at Massachusetts General Hospital, at Harvard Medical School. Dr. Cudkowicz completed medical training at the Health Science and Technology program of Harvard Medical School, and she was a resident in Neurology at Massachusetts General Hospital. She obtained a Master's degree in Clinical Epidemiology from the Harvard School of Public Health in June, 1996. She was also a fellow in the Massachusetts General Hospital (MGH)/ Massachusetts Institute of Technology (MIT) Clinical Investigator Training Program from 1994 to 1996.

Dr. Cudkowicz co-directs the MGH Amyotrophic Lateral Sclerosis (ALS) clinic and the Neurology Clinical Trials Unit (NCTU) at MGH and Partners Healthcare System. She co-directs the Northeast ALS Consortium with Dr. Jeremy Shefner. She is on the medical advisory board for the Muscular Dystrophy Association and the Massachusetts Chapter of the Amyotrophic Lateral Sclerosis Association.

 

Julaine Florence, PT, DPT

Dr. Florence is Associate Professor in the Department of Neurology at Washington University School of Medicine, St. Louis, MO. She presently serves as Director of Clinical Studies for the Neuromuscular Division. Her training was at Washington University School of Medicine and includes a bachelors in Physical Therapy, masters in Health Science and doctorate in Physical Therapy.

Dr. Florence has been active in the design, implementation and documentation of the reliability, sensitivity and validity of outcome measures and endpoints in the assessment of individuals with neuromuscular diseases as well as serving as a clinical evaluator or coordinator in multiple therapeutic trials. Besides her activity in clinical trials Dr. Florence is on faculty at the WU Program in Physical Therapy and serves as a consultant on outcome measure design and implementation to a variety of academic institutions, biotech and pharmaceutical companies.

 

Terry Heiman-Patterson, MD

 

Jeffrey Rothstein, PhD, MD

Dr. Jeffrey Rothstein is currently Professor of Neurology and Neuroscience in the Department of Neurology at Johns Hopkins University. He is also Director for the Robert Packard Center for ALS Research at Johns Hopkins and co-Director of the ALS clinic at Johns Hopkins. He received his medical training, and PhD and research training at the University of Illinois and the University of Chicago. He trained in Neurology and Neuromuscular disease at Johns Hopkins University.

Dr. Rothstein is a neurologist and neurobiologist with a major commitment to investigations of mechanisms of neurodegeneration. He has been a strong proponent of the roles of excitotoxicity, mitochondrial dysfunction, protein aggregation, and oxidative stress in the pathogenesis of ALS. More specifically, he has proposed that abnormalities in glutamate transport contribute to the pathogenesis of cellular abnormalities that occur in motor neuron death. His laboratory studies the basic biology of membrane transporter, the cellular events that underlie regulation of transporters, and the development of novel drug- and cellular-based therapeutics targeted for both transporters and motor neuron death. He has developed both in vitro and in vivo models of motor neuron degeneration.

 

David Schoenfeld, PhD

Dr. Schoenfeld is a Professor of Biostatistics at Massachusetts General Hospital and Harvard Medical School, and Director of the Biostatistics Center at Massachusetts General Hospital. The Biostatistics Center at MGH provides statistical support for investigators conducting clinical and laboratory research. He is the lead statistician for the MGH General Clinical Research Center (GCRC), and collaborates extensively with neurologists. He is principal investigator for the Clinical Coordination Center for the ARDS Network, which represents over 30 hospitals and conducts multicenter clinical trials on Acute Respiratory Distress Syndrome.

Dr. Schoenfeld developed the first omnibus goodness of fit test for the proportional hazards regression model, a model that is used extensively in clinical trials, which have survival or time to progression as an endpoint. He also developed widely used graphical techniques for this model. Dr. Schoenfeld’s current research involves the application of causal inference to clinical trials and methods for the analysis of studies involving gene arrays.

Dr. Schoenfeld developed the first omnibus goodness of fit test for the proportional hazards regression model, a model that is used extensively in clinical trials which have survival or time to progression as an endpoint. He also developed widely used graphical techniques for this model. Dr. Schoenfeld’s current research involves the application of causal inference to clinical trials and methods for the analysis of studies involving gene arrays.

 

Jeremy Shefner, MD, PhD

Dr. Shefner is Professor and Chair of Neurology at Upstate Medical University, and Director of the Clinical Neurophysiology Laboratory and the MDA/ALS Research and Treatment Center at University Hospital, in Syracuse, New York. He is trained in neurology and clinical neurophysiology, and has devoted his clinical and research efforts to ALS. He received a PhD in experimental psychology from the University of Illinois in 1976, and was a NRSA Research Fellow from 1976 to 1979. He completed a fellowship in clinical neurophysiology and neuromuscular disease with the Brigham and Women's Hospital in Boston, Massachusetts. While at the Brigham and Women's Hospital, Dr. Shefner directed the ALS Clinic and participated in multiple industry sponsored ALS clinical trials. Since moving to Syracuse, he established the multidisciplinary ALS clinic there, and continues to participate in both industry- and investigator-initiated clinical trials.

Dr. Shefner has also been active in the development of a physiological outcome measure in trials of ALS. This outcome measure, called Motor Unit Number Estimation (MUNE) has been used in studies of both animals and patients with ALS.

 

Rup Tandan, MD, FRCP

Dr. Tandan is Professor and Vice Chairman in the Department of Neurology, at the University of Vermont College of Medicine in Burlington, Vermont. He serves as the Director of the Neuromuscular Disorders Section, the ALS Clinical and Research Center, the Muscular Dystrophy Association Clinic, and the International Postpolio Foundation Clinic at Fletcher Allen Health Care and the University of Vermont College of Medicine. He received his medical degree from King George's Medical College at the University of Lucknow, India. He completed residencies in Internal Medicine in England, in Neurology at the University of Birmingham, England, and at the University of Michigan, Ann Arbor. He trained as a Fellow in Clinical Electromyography and Neuromuscular Diseases at the University of Michigan, as a Neuromuscular Fellow at the University of Vermont College of Medicine.

Dr. Tandan is a Fellow of the Royal College of Physicians of the United Kingdom, and a Fellow of the American Board of Psychiatry and Neurology. He is on the Medical Advisory Board of the National ALS Association, and serves as Chairperson of the Medical Directors of the National ALS Association ALS centers. He has served as Principal Investigator of many studies, and has collaborated in several others. His current research interests are clinical trials and the study of energy metabolism in ALS using stable isotrope technology. He has also written and co-written over 150 scientific abstracts and articles.

 

NEALS Coordination and Data Management Centers

The Coordination and Data Management Centers for the Northeast ALS Consortium are located in Neurology Clinical Trials Unit (NCTU) at the Massachusetts General Hospital (MGH.) The MGH Neurology Clinical Trials Unit was formed in 1994 to conduct clinical research in ALS and other neurological disorders. The NTCU has helped investigators in the department conduct clinical trials in a variety of neurological disorders including migraine, diabetic neuropathy, stroke, multiple sclerosis, ALS and Huntington’s Disease.

The NCTU was the Coordination and Data Management Center for three clinical drug trials in ALS: a safety and efficacy study of topiramate, a clinical trial of conenzyme Q10, and a clinical trial of Celebrex. The NCTU will be the Coordination and Data Management Center for the trial of sodium phenylbutyrate in ALS and the trial of Ceftriaxone in ALS. The NCTU coordinates clinical trials in Huntington’s Disease and Cerebral Amyloid Angiopathy. Dr. Cudkowicz and the MGH NCTU also perform non-drug studies, including studies of ALS biomarkers. Under the direction of Dr. Merit Cudkowicz, the NCTU staff consists of a Nurse Coordinator, three Clinical Research Coordinators, four Project Managers, a Director of Strategic Development, two Data Managers, a Data Entry Specialist, a director of finance and an administrator.

 

NEALS Clinical Trial Centers

Institution	Location	Investigator(s)
Baylor College of Medicine	Houston, TX	Yad Harati, MD
Baystate Medical Center	Springfield, MA	George Baquis, MD and Carmel Armon, MD
Beth Israel Deaconess Medical Center	Boston, MA	Elizabeth Raynor, MD and Seward Rutkove, MD
Beth Israel Medical Center-Albert Einstein College	New York, NY	Stephen N. Scelsa, MD
Boston University Medical Center	Boston, MA	Janice Wiesman, MD
Brigham and Women's Hospital	Boston, MA	Anthony Amato, MD
Carolinas Medical Center	Charlotte, NC	Elena Bravver, MD
Cedars-Sinai Medical Center	Los Angeles, CA	Abirami Muthukumaran, MD
Cleveland Clinic Foundation	Cleveland, OH	Erik Pioro, MD, PhD
Columbia University	New York, NY	Hiroshi Mitsumoto, MD and Paul Gordon, MD
Cornell University	New York, NY	M. Flint Beal, MD, Science Advisor
Dartmouth Hitchcock Medical Center	Lebanon, NH	Jeffrey Cohen, MD
Drexel University	Philadelphia, PA	Terry Heiman-Patterson, MD and Anahita Deboo, MD
Duke University	Durham, NC	Richard Bedlack, MD, PhD
Emory University	Atlanta, GA	Jonathan Glass, MD, Michael Benatar, MD and Dr. Jaffar Khan
George Washington University	Washington, DC	Elliam Bayat, MD
Hennepin County Medical Center	Minneapolis, MN	Ezgi Tiryaki, MD
Henry Ford Hospital	Detroit, MI	Daniel Newman, MD
Indiana University	Indianapolis, IN	Robert Pascuzzi, MD
Johns Hopkins University	Baltimore, MD	Jeffrey Rothstein, MD, PhD and Nicholas Maragakis, MD
Lahey Clinic	Burlington, MA	James Russell, DO
Massachusetts General Hospital	Boston, MA	Merit Cudkowicz, MD, MSc
Mayo Clinic, Jacksonville	Jacksonville, FL	Kevin Boylan, MD
McGill University-Montreal Neurological Institute	Montreal, Quebec, Canada	Angela Genge, MD
Medical College of Wisconsin-Froedtert Memorial Lutheran Hospital	Milwaukee, WI	Paul Barkhaus, MD
Medical University of South Carolina	Charleston, SC	David E. Stickler, MD
Methodist Neurological Institute	Houston, TX	Stanley Appel, MD and Ericka Simpson, MD
Mt. Sinai Hospital	New York, NY	Dale Lange, MD
Ohio State University	Columbus, OH	John T. Kissel, MD
Oregon Health & Science University	Portland, OR	Jau-Shin Lou, MD, PhD
Pennsylvania State University	Hershey, PA	Zachary Simmons, MD
Phoenix Neurological Associates-Samaritan ALS Clinic	Phoenix, AZ	Todd Levine, MD and David Saperstein, MD
Providence ALS Center	Portland, OR	Kimberly Goslin, MD
Rhode Island Hospital	Providence, RI	George Sachs, MD, PhD
Robert Wood Johnson Medical School/UMDNJ	New Brunswick, NJ	Jerry Belsh, MD
Saint Louis University	Saint Louis, MO	Ghazala Hayat, MD
Stanford University	Stanford, CA	S. Charles Cho, MD
State University of New York, Stony Brook	Stony Brook, NY	Rahman Pourmand, MD
SUNY Upstate Medical University	Syracuse, NY	Jeremy Shefner, MD, PhD
Texas Neurology, PA	Dallas, TX	Daragh Heitzman, MD
University of Arkansas	Little Rock, AR	Stacy Rudnicki, MD
University of British Columbia-Vancouver General Hospital	Vancouver, BC, Canada	Hannah Briemberg, MD
University of California Irvine	Orange, CA	Tahseen Mozaffar, MD
University of California Los Angeles	Los Angeles, CA	Michael Graves, MD
University of California San Diego	La Jolla, CA	Nayan P. Desai, MD and Don Cleveland, PhD
University of California San Francisco	San Francisco, CA	Catherine Lomen-Hoerth, MD, PhD
University of Chicago	Chicago, IL	Kourosh Rezania, MD
University of Colorado	Aurora, CO	Bjorn Oskarsson, MD
University of Connecticut	Farmington, CT	Kevin Felice, DO
University of Illinois at Chicago	Chicago, IL	Julie Rowin, MD
University of Kansas	Kansas City, Kansas	Richard Barohn, MD
University of Kentucky	Lexington, KY	Edward Kasarskis, MD, PhD
University of Miami	Miami, FL	Khema Sharma, MD and Ashok Verma, MD
University of Nebraska	Lincoln, NE	Gary Pattee, MD
University of Nevada	Henderson, NV	David L. Ginsburg, MD
University of Pittsburgh	Pittsburgh, PA	David Lacomis, MD and Sasa Zivkovic, MD
University of Puerto Rico	San Juan, PR	Carlos A. Luciano, MD
University of Texas Health Science Center	San Antonio, TX	Carlayne Jackson, MD
University of Toronto	Toronto, Ontario	Lorne Zinman, MD, MSc, FRCPC
University of Utah	Salt Lake City, UT	Mark Bromberg, MD
University of Vermont	Burlington, VT	Rup Tandan, MD, FRCP
University of Virginia	Charlottesville, VA	Lawrence H. Phillips, II, MD and Ted Burns, MD
University of Western Ontario, London Health Sciences Centre	London, Ontario, Canada	Christen Shoesmith, MD
Upstate Neurology Consultants, LLP	Albany, NY	James Wymer, MD
Vanderbilt University	Nashville, TN	Peter D. Donofrio, MD
Virginia Mason Medical Center	Seattle, WA	John M. Ravits, MD, FAAN
Wake Forest University	Winston-Salem, NC	James Caress, MD
Wayne State University	Detroit, MI	Richard A. Lewis, MD
Washington University	St. Louis, MO	Alan Pestronk, MD
West Virginia University	Morgantown, WV	Laurie Gutmann, MD
Wilmington Health Associates	Wilmington, NC	Susan Torres, MD
Yale University	New Haven, CT	Jonathan Goldstein, MD and Steven Novella, MD