Member Portal    
ALS Trials
Browse Recruiting Trials
Search for a Trial
Clinical Trials 101
Questions about Clinical Research?
Contact an ALS Trial Liaison

Call or email us with your questions
about clinical research

(877) 458-0631

A Phase I, Open-label, First-in-human Feasibility and Safety Study of Human Spinal Cord derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis

Study Focus:
The purpose of the study is to determine the feasibility, safety, and toxicity of human spinal cord-derived neural stem cell transplantation for the treatment of amyotrophic lateral sclerosis.
Amyotrophic Lateral Sclerosis (ALS), Sporadic ALS, Familial ALS
Study Category:
Stem cell trial, Device
Study Status:
Study Results:
Phase I
Interventional Trial
with active agents/drugs only
Funding Source:
Neuralstem Inc.
Study Chair(s)/Principal Investigator(s):
Eva Feldman, MD, PhD (University of Michigan) ID:
NEALS Affiliated?
Study Summary:
Previous research has shown that on autopsy, ALS patients are found to have increased levels of the amino acid glutamate accumulated in the brain and spinal cord. This increase is thought to be caused by a decrease in the glutamate transporter which normally "cleans up" glutamate from the cells. Human spinal cord-derived neural stem cells (HSSC) are known to express amino acid transporters and it is hoped that this action will reduce the toxicity of accumulated glutamate and benefit ALS patients. A second hypothesized benefit of HSSC is their ability to secrete neurotrophic support factors. Neurotrophic factors support the health of nerves.

These stem cells have been engineered from the spinal cord of a single fetus electively aborted after eight weeks of gestation. The tissue was obtained with the mother's consent. The cells are transplanted into the ALS subject's spinal cord after laminectomy, an operation that removes bone surrounding the spine. After the spinal cord is exposed, a device manufactured for this purpose will be mounted onto the subject and will hold a syringe filled with the cells. The syringe will have a needle attached and the needle will enter the spinal cord at 5-10 locations injecting the cells. The device will minimize trauma to the spinal cord caused by the needle by making the punctures precise and steady, and by injecting the material at a slow and steady speed.

This study includes five groups of ALS subjects. The first three groups, A, B and C, have completed enrollment. The first group (A) included six subjects and the remaining groups have three subjects each. The groups have slightly different inclusion criteria and receive different procedures. The first group (A) included patients with advanced disease who were unable to walk. This group (A) included both ALS patients with the ability to breathe on their own as well as those with tracheostomies and ventilators. The first three subjects in group A underwent a laminectomy and received transplant injections of HSSC on one side of their lower spinal cord. The second three subjects in the first group (A) received transplant injections of HSSC on both sides of their lower spinal cord. Group B included ALS patients who were still ambulatory. They received transplant injections of HSSC on one side of their lower spine. Group C included ALS patients who were still ambulatory and received transplant injections on both sides of their lower spine. Group D patients will be ambulatory with some arm dysfunction and will receive transplant injections on one side of their neck. Lastly group E will include ALS patients who are ambulatory and will receive both injections on one side of their neck and injections on both sides of their lower spine. Because the HSSC are human in origin, subjects will receive immunosuppressive medications to prevent the rejection of the cells. Right before and immediately after surgery subjects will receive infusions of a drug called basiliximab. After surgery they will take prednisone and be tapered off that medication over one month. They will also be given two other immunosuppressive agents, tacrolimus and mycophenolate mofetil after surgery and it is expected that the subjects will take these drugs for the duration of their lives.
Participant Duration:
4 years + 3 months active study participation; lifetime follow-up
# of Subjects:
Enrollment Start Date:
Enrollment End Date:
Posting Last Modified Date:
Date Study Added to
More information:
Enrolling BY INVITATION. Please click on "Site Contact Information" below for Emory contact information to learn more about enrollment in this trial.
Eligibility Criteria
Male & Female
Minimum Age:
Min Vital Capacity (% predicted normal):
Time since Symptom Onset:
Not applicable
Time since Diagnosis:
Not applicable
Can participants use Riluzole?
Coordinating Center
Contact Information
Elene McLoughlin
Massachusetts General Hospital
149 13th Street
Charlestown, Massachusetts 02129
Other Eligibility Criteria:
If taking riluzole, dose must be stable for three months; geographic accessibility to the site is required (Atlanta, GA); subjects must have a willing and able caregiver; have no unstable medical conditions and be medically able to tolerate the surgery and immunosuppressive medications.

Site Contact Information

 (Please click to show/hide)
For Patients & Families
Educational Webinars
Online NEALS Resources
Other Resources
Contact a Disease Specialist
Clinical Research Blog

For Researchers
Become a NEALS member
Post an ALS Clinical Trial
NEALS CRO Capabilities
Annual NEALS Meeting
NEALS Samples
NEALS Historical Data
ALS Trials
Browse Recruiting Trials
Search for a Trial
Clinical Trial News
Clinical Trials 101

Get Involved
Donate samples
Donate to our Clinical Research
About Us
Our Mission
Our Structure
Our Research
NEALS Members
Motor Neuron Disease
Connect with us: Member Portal       Donate Now!       Help       Sitemap       Contact Us