- Additional data from the completed phase 1 trial of Neuralstem’s NSI-566 neural stem cells show that trial participants who had limb-onset (as opposed to bulbar-onset) ALS and who received the cells earlier rather than later in their disease course had better outcomes.
- The new data from the phase 1 trial were reported May 17, 2013; trial results reported in December 2012 showed the stem cells and the method used to transplant them were safe and well-tolerated.
- A phase 2 trial of Neuralstem’s NSI-566 neural stem cells has been approved by the U.S. Food and Drug Administration.
Recently announced data from a phase 1 clinical trial of NSI-566 neural stem cells in people with amyotrophic lateral sclerosis (ALS) show that a subset of trial participants had better outcomes than the others.
Neural stem cells generate motor neurons and glia. Biotherapeutics company Neuralstem, developer of the NSI-566 cells, is studying whether they can improve respiratory function and prolong life span in ALS.
Previously reported results from the completed phase 1 trial showed that the stem cells and the method used to transplant them were safe and well-tolerated.
The additional data were reported May 17, 2013, by trial principal investigator Eva Feldman at a conference in Romania, as well as via a May 20, 2013, Neuralstem press release.
Limb-onset, early treatment correlated with better outcomes
According to Feldman, new analysis showed that six trial participants, now approximately 700 to 850 days post-surgery, “have a stable, very slowly progressing or improved disease course.”
All six share two characteristics: They have limb-onset ALS (as opposed to bulbar-onset ALS, which is known to progress more rapidly); and they received stem cell transplantation early (as opposed to later) in the course of their disease (an average two years, one month after onset of symptoms). Two of the six participants who showed stabilization or improvement were among those who received stem cell transplants in both the lower and upper back regions of their spinal cords.
Of the nine remaining participants, three have had little change in the trajectory of their disease, and six have died. The three who have had no change had longstanding disease (5.6, 11.6 and 12.7 years) at the time of transplantation.
The six who died (seven to 30 months after receiving the neural stem cells) had bulbar symptoms at the time of their transplantation.
Earlier reports showed safety, tolerability
Jonathan Glass, director of the MDA/ALS Center at Emory University School of Medicine in Atlanta, presented results from the trial at the 23rd International Symposium on ALS/MND, held in Chicago Dec. 5-7, 2012.
Glass reported that the stem cells and the surgical transplant method were safe and well-tolerated in all trial participants, with evidence of a treatment effect — an interruption of the progression of the disease — in some. In addition, trial investigators were able to detect transplanted stem cells in the autopsies of six trial participants who had died (five of ALS progression and one of unrelated heart failure).
Phase 1 trial followed a ‘risk-escalation’ format
The phase 1 trial — the 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 (FDA) 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 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.
Phase 2 dose-escalation trial approved by FDA
Neuralstem announced in April that it had received FDA approval to conduct a phase 2 clinical trial of its NSI-566 stem cells in people with ALS.
The trial, designed to assess safety and determine the maximum tolerated dose, will expand to include a site at the University of Michigan Health System in Ann Arbor, as well as the original site at Emory University in Atlanta.
For more on the phase 2 trial, see Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis, or search for Trial ID NCT01730716 at ClinicalTrials.gov.
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 ClinicalTrials.gov.