Neuralstem Phase 1 Stem Cell Trial Shows Safety, Possible Efficacy

by Amy Madsen on Wed, 2012-04-04 06:00

Transplantation of stem cells into the spinal cords of 12 people with ALS has proven safe and well-tolerated, with no long-term complications

Article Highlights:
  • Results from a phase 1 study in ALS to evaluate the safety of stem cells transplanted into the lumbar (lower back) region of the spinal cord, as well as the transplantation technique itself, showed the treatment to be safe and well-tolerated.
  • One trial participant showed clinical improvement, even though the trial was not designed to demonstrate efficacy.
  • Maryland biotherapeutics company Neuralstem announced March 28, 2012, that the results, taken from the first 12 patients with ALS, have been reported online in the journal Stem Cells. Testing continues to progress through increased levels of risk.

Neuralstem's spinal cord stem cells, and the surgical technique used to transplant them, proved to be safe and well-tolerated in a phase 1 clinical trial in 12 people with amyotrophic lateral sclerosis (ALS). Although the trial wasn't designed to show efficacy, one participant showed signs of clinical improvement.

This first U.S.-based trial of neural stem cells in ALS opened at the MDA/ALS Center at Emory University in January 2010 and is designed to progress through increased levels of risk. (Note: MDA is not funding Neuralstem for this trial.)

The company's goal is to determine whether the cells can improve respiratory function and prolong life span in ALS.

About the trial

All of the initial 12 trial participants received injections of stem cells in the lumbar (lower back) region of the spinal cord. The first six were nonambulatory (unable to walk), with permanent paralysis; the second set of six still had the ability to walk. In each group of six participants, the first three received five injections on one side of the spinal cord, and the second set of three received 10 injections (five on each side of the spinal cord).

Based on positive interim safety results, the FDA granted Neuralstem approval to begin giving injections to people with ALS in the cervical (neck) region of the spine. On March 7, 2012, Neuralstem announced that the 14th patient in the trial has now received stem cells. She is the second participant to receive cells in the cervical region of the spinal cord and the first woman to participate in the trial.

Neuralstem, of Rockville, Md., announced in a March 28, 2012, press release that the results taken from the first 12 trial participants have been reported online in the journal Stem Cells. Stem Cells is a peer-reviewed journal, which means articles that appear in it must be approved for publication by an independent panel of experts in the field of study. Publication in such a journal can be an important step in moving an experimental treatment through the stages of therapy development. 

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 ClinicalTrials.gov.

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