The journal Stem Cells has released an online version of a paper ahead of the print version that describes an important experiment in the treatment of amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehring’s disease. This paper describes an experiment that resulted from collaboration between the University of Michigan, Emory University and NeuralStem, Inc., which sponsored the study.
In this clinical trial, 12 patients were transplanted with spinal cord stem cells. All transplantations were done at Emory University. The early results of this trial show that spinal stem cells can be safely delivered into the spinal cords of ALS patients. This study might certainly open the door to further research on stem cell-based treatments for ALS.
All 12 patients had ALS and none experienced any long-term complications from this stem cell transplantation procedure. Additionally, none of the patients showed any signs of rejecting the implanted cells. Because inflammation in the spinal cord accelerates the progression of the disease, there were concerns that the implantation could increase the disease in these patients. However, in the months following the surgery that was used to inject the stem cells, none of the patients showed evidence that their ALS progression was accelerating.
Eva Feldman, M.D., Ph.D. is the principal investigator at the University of Michigan Medical School for this trial and serves as a consultant to NeuralStem. She is also the director of the A. Alfred Taubman Medical Research Institute and the U-M Health System’s ALS Clinic. Dr. Feldman stated, “This important publication reinforces our belief that we have demonstrated a safe, reproducible and robust route of administration into the spine for these spinal cord neural stem cells. The publication covers data up to 18 months out from the original surgery. However, we must be cautious in interpreting this data, as this trial was neither designed nor statistically powered to study efficacy.”
The trial began in January 2010 at Emory University. The first 12 patients received neural stem cell transplants in the lumbar, or lower, region of the spinal cord. After reviewing safety data from these patients, the Food and Drug Administration granted approval for the trial to advance to the final two groups of patients (three in each group), all of whom will be transplanted in the cervical, or upper, region of the spinal cord.
Nicholas Boulis, M.D., associate professor of neurosurgery at Emory School of Medicine, performs the surgery that implant the neural stem cells. Boulis also developed the device he used inject the stem cells into the spinal cord. This same device received a notice of patent allowance from U.S. Patent and Trademark Office in October. NeuralStem has purchased an exclusive license to this technology. Boulis trained in neurosurgery at University of Michigan and collaborated on research with Feldman during his seven years of residency. He holds an adjunct associate professor of neurology position at University of Michigan and is one of the Taubman Scholars at the U-M Taubman Institute.
This clinical trial is one of the first U.S. clinical trials of stem cell injections into the spinal cord for the treatment of ALS. NeuralStem, Inc., a Maryland-based company, is funding the clinical trial and has also provided the human neural stem cells for transplantation. NeuralStem’s cells have the ability to mature into various types of cells in the nervous system, including the motor neurons that are specifically lost in ALS. However, scientists say the goal of stem cell transplantation is not to generate new motor neurons, but to protect the still-functioning motor neurons by nurturing them with the stem cells, and therefore, potentially slowing the progression of the disease.