- Neuralstem is seeking U.S. Food and Drug Administration approval to expand the design of its phase 1 stem cell safety trial to include endpoints that will allow investigators to assess the cells' efficacy.
- The company also has asked the FDA for permission to increase both the dosage and the total number of people allowed to take part in the trial, which originally was designed to test the company's neural stem cells in 18 people with ALS.
- The company announced that the FDA has granted approval for three people with ALS who participated in an earlier stage of the phase 1 trial to return for a later stage.
The biotherapeutics company Neuralstem is seeking to amend the design of its ongoing phase 1 stem cell trial in amyotrophic lateral sclerosis (ALS) to include evaluations of efficacy (how well the therapy works).
Although the phase 1 trial isn’t currently designed to evaluate efficacy, early results reported in March noted that one participant showed signs of clinical improvement.
The Maryland-based company also is seeking permission to increase the treatment dose and the total number of trial participants (originally set at 18).
Any amendments to the trial design must be approved by both the U.S. Food and Drug Administration (FDA) and the institutional review board at Emory University, where the trial is being held.
The company announced last week that the FDA has granted approval for three people who were treated in an earlier stage of the trial to participate again in the next stage of this trial.
Early results show safety
The trial, which opened at the MDA/ALS Center at Emory University in January 2010, is the first U.S.-based trial of neural stem cells in ALS. Neural stem cells generate neurons and glia (a type of motor neuron support cell) in the brain. The company's goal is to determine whether these cells can improve respiratory function and prolong life span in ALS. (Note: MDA is not funding Neuralstem for this trial.)
Interim results, published online March 12, 2012, in Stem Cells, showed that Neuralstem's neural stem cells, and the surgical technique used to transplant them, were safe and well-tolerated in 12 people with ALS. (Abstract available at no cost; complete article available for purchase with subscription.) All of the 12 received injections of stem cells in the lumbar (lower back) region of the spinal cord. The first six (Group A) were nonambulatory (unable to walk), with permanent paralysis; the second set of six (Groups B and C) still had the ability to walk.
Since then, three additional ambulatory trial participants (Group D) have received injections to the cervical (neck) region of the spinal cord.
Three may return for next stage of trial
Three trial participants in Group C, who received their first set of injections 15 to 17 months ago, will be allowed to participate in the next stage of the trial if they meet inclusion criteria.
The next stage is set to include three people with ALS who will receive a total of 15 injections: five on each side of the spinal cord in the lumbar region, and five in the cervical region. Returning participants, who already have received 10 injections into the lumbar region, will only receive five injections in the cervical region.
"The return of these patients to the trial for second treatments is a continuing validation of the trial's safety," Neuralstem Chairman and Chief Scientific Officer Karl Johe said in a press release.
"Treating these patients who have already received injections in one part of their spine allows us to both increase the overall dosage for each patient as well as transplant them in regions of the spine where they have not been treated," Johe noted. "This next cohort of patients will be the first in the world to receive stem cell transplants in both cervical and lumbar regions of their 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 in the search box at ClinicalTrials.gov.