- Neuralstem has received approval from the U.S. Food and Drug Administration to conduct a phase 2 trial of its NSI-566 neural stem cells, a treatment aimed at improving respiratory function and prolonging life in people with amyotrophic lateral sclerosis.
- The dose-escalation and safety trial will take place at the MDA/ALS Center at Emory University in Atlanta, and at the ALS Clinic at the University of Michigan Health System in Ann Arbor.
- The cells and the surgical method used to transplant them proved to be safe and well-tolerated in people with ALS in an earlier phase 1 trial.
Maryland biotherapeutics company Neuralstem today announced that it has received approval from the U.S. Food and Drug Administration (FDA) to conduct a phase 2 clinical trial to test its NSI-566 neural stem cells in people with amyotrophic lateral sclerosis (ALS).
Neural stem cells generate muscle-controlling nerve cells (motor neurons) and glia (a type of motor neuron support cell) in the brain. It's hoped the experimental therapy will improve respiratory function and prolong life in ALS.
The trial, which is designed to assess safety and determine the maximum-tolerated dose, will expand to two centers:
- the MDA/ALS Center at Emory University in Atlanta (where an earlier phase 1 trial was conducted), with principal investigator Jonathan Glass; and
- the ALS Clinic at the University of Michigan Health System in Ann Arbor, with principal investigator Eva Feldman.
Determining the maximum-tolerated dose
Investigators expect to enroll 15 participants, who will be divided into five different dosing groups. The first 12 participants will receive stem cell injections into the cervical (neck) region of the spinal cord. The last three participants will receive injections in both the cervical and lumbar (lower back) regions of the spinal cord.
In addition to other eligibility criteria, participants must be ambulatory, and must live within close geographic proximity to the research center at which they will participate.
"The aim of this phase 2 trial is to obtain the maximum-tolerated dose using the same route of administration as in phase 1, which was through direct injections into the gray matter of the spinal cord," Neuralstem Chairman and Chief Scientific Officer Karl Johe said in an April 17, 2013, Neuralstem press release. "In phase 1, we started with just five injection sites per patient, and advanced to a maximum of 15 injections of 100,000 cells each. In phase 2, we will advance up to a maximum of 40 injections, and 400,000 cells per injection based on safety."
Building on encouraging phase 1 results
The new phase 2 trial follows a completed phase 1 trial in which investigators successfully completed 18 stem cell transplants in 15 trial participants. (Three participants who were treated earlier in the trial were allowed by the FDA to return later in the trial.)
The first 12 participants each received neural stem cell injections to the lumbar 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 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.
Results from that trial showed that the cells and the surgical technique used to transplant them were well-tolerated and that the cells survived long term. In addition, the experimental therapy appeared to have interrupted disease progression in a subgroup of participants. For more, read Neuralstem Completes Stem Cell Trial.
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.
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.