- The responses of the first six participants who received injections of neural stem cells into their spinal cords in a phase 1 trial in ALS have been judged to demonstrate adequate safety, allowing the trial to proceed.
- The next trial participants to be injected will be able to walk at enrollment, in contrast to the first group, who had lost this ability prior to enrollment.
- The trial is designed to assess safety and feasibility of spinal-cord injections of neural stem cells but is not expected to benefit the participants.
- The stem cells are derived from a fetal spinal cord and were developed by the trial's sponsor, the biotherapeutics company Neuralstem.
The Maryland biotherapeutics company Neuralstem will move its clinical trial of neural stem cells in people with amyotrophic lateral sclerosis (ALS) into its next stage, following a thumbs-up from the trial's safety monitoring board, the company announced Oct. 18, 2010. (See Neuralstem Updates ALS Clinial Trial Progress.)
About the Neuralstem trial
This phase 1 trial, designed mainly to assess the safety and feasibility of injecting neural stem cells into the spinal cords of people with ALS, is being conducted at Emory University in Atlanta, under the supervision of neurologists Eva Feldman and Jonathan Glass. Glass directs the MDA/ALS Center at Emory, but MDA is not funding this trial.
The cells are derived from the spinal cord of a human fetus. In Neuralstem's laboratories, the cells been made to multiply and create a supply for patients in research studies. Both the cells and the procedure for injecting them are experimental.
This first U.S.-based trial of neural stem cells in ALS opened in January 2010. (See Stem cell trial at Emory on track and First U.S. trial of neural stem cells in ALS gets FDA green light.)
As of Oct. 18, 2010, six participants had received injections of stem cells into their spinal cords. All six had lost the ability to walk prior to entering the study. The first three people each received five injections into one side of the lumbar (lower back region) of the spinal cord, and the next three each received 10 injections into both sides of the lumbar cord. The trial's safety board did not see any warning flags with this stage of the trial.
The next stage will involve injecting six more people with ALS who are at an earlier stage of the disease than the first group and who still will be walking at study entry. The first three in this group each will receive five injections into one side of the lumbar cord, and the next three will receive injections into both sides of the lumbar cord.
A final stage is planned, in which six additional trial participants will receive injections into the cervical (neck) region of the spinal cord.
Trial participants must live near Emory University Hospital and meet many other study criteria. Extensive testing, such as ultrasound exams, respiratory evaluations, MRI scans and strength measurements will be performed at intervals during the course of this multiyear study. Participants will need to take immunosuppressive medications, probably for life, to help them tolerate the transplanted cells.
Complete information about the trial and details about participation, including consent forms, can be viewed at Emory ALS Center Stem Cell Trial.
Meaning for people with ALS
Benefit to people with ALS is not expected from this trial, although a great deal about the injection procedure and the behavior of the injected cells in the body is likely to be learned.
The ultimate goal of stem cell therapy in ALS is for the injected cells to become functioning nerve cells that will substitute for those lost in ALS, or for the injected cells to develop into nervous-system support cells that can help preserve remaining nerve cells. In either case, the hope is that stem cells may someday stabilize the disease course in ALS or possibly even reverse some of the disease-associated functional losses.
The Neuralstem trial poses several risks, among them the possibility that the immune system could reject the new cells, potentially harming trial participants; that the immunosuppressive medications required to tolerate the injected cells could cause serious problems; and that the spinal cord could be damaged by injections.
However, the positive safety review for the first six participants in this trial is good news for the ALS community. Stem cell trials that might eventually benefit patients cannot proceed without this vital first step.