Neural Stem Cell Trial in ALS ‘Very Successful’ So Far

by Amy Madsen on Fri, 2012-10-12 10:53

Data from Neuralstem’s completed phase 1 trial of neural stem cells in ALS showed safety and tolerability in all, evidence of a ‘treatment effect’ in some

Article Highlights:
  • Neuralstem's NSI-566 neural stem cells and the surgical method used to transplant them proved to be safe and well-tolerated in people with ALS in a phase 1 trial.
  • In addition, data from the completed trial shows that the experimental therapy appears to have interrupted disease progression in some trial participants.
  • Neuralstem is looking into additional clinical trials that will allow for the study of larger doses of the drug and assess possible therapeutic effect.  

Results from a completed phase 1 trial of neural stem cells in people with amyotrophic lateral sclerosis (ALS) show that the stem cells and the surgical method used to transplant them were safe and well-tolerated. In addition, the experimental therapy appears to have "interrupted the progression of the disease" in some trial participants.

The new findings follow treatment of the last trial participant in August 2012 and confirm interim data reported while the study was still in progress.  

Rockville, Md.-based biotherapeutics company Neuralstem, developer of the NSI-566 neural stem cells, plans to determine whether the cells can improve respiratory function and prolong life span in ALS. Neural stem cells generate muscle-controlling motor neurons and glia (a type of motor neuron support cell) in the brain.

"We believe we are seeing evidence of a treatment effect in some patients over a sustained period of time," Karl Johe, chairman of the board and chief scientific officer of Neuralstem, said in a press release. Johe said more clinical trials are needed to study larger doses of the drug and to assess its possible therapeutic effect.

The trial followed a ‘risk escalation’ format

This first U.S.-based trial of spinal cord stem cells in ALS opened at the MDA/ALS Center at Emory University in January 2010 and followed a "risk escalation" format.  

Fifteen trial participants received a total of 18 neural stem cell treatments. (Three participants who were treated in an earlier phase were allowed by the U.S. Food and Drug Administration to return later in the trial.)

The first 12 participants each received neural stem cell injections to the lumbar (lower back) region of the spine, with treatment being administered first to those who were nonambulatory (had lost the ability to walk) and then to those who still were ambulatory.

The trial then advanced to transplantation in the cervical (upper back) region of the spine. Three participants received injections in the cervical region only. The last three participants received injections in the cervical region in addition to the lumbar injections they had received earlier in the trial.

Principal investigator Eva Feldman announced the new results at the American Neurological Association annual meeting in Boston, Oct. 8, 2012. Neuralstem also plans to present preclinical data on the NSI-566 neural stem cell line at the 42nd Annual Meeting for the Society of Neuroscience, Oct. 13-17, 2012, in New Orleans.

Neuralstem's patented technology allows it to produce commercial quantities of neural stem cells for the human brain and spinal cord.

For more information on this trial, see Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of ALS; or enter NCT01348451 into the search box at


About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

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