Stem Cell Injections in Lou Gehrig's Disease can be Given Safely, New Research Shows


tANN ARBOR — The first published results from an early-stage clinical trial show that spinal cord stem cells can be delivered safely into the spines of patients with the condition commonly known as Lou Gehrig's disease, opening the door for further research on this innovative approach.

tIn a paper published online ahead of print publication in the peer-reviewed journal Stem Cells, a team from the University of Michigan, Emory University and study sponsor NeuralStem, Inc. report the results from 12 patients who took part in a study being conducted at Emory.

tAll had amyotrophic lateral sclerosis, the inevitably fatal degenerative disease of the nervous system that many call Lou Gehrig's disease. None experienced any long-term complications related to either the surgical procedure or the implantation of stem cells, or showed signs of rejecting the cells. And in the months following the surgery to inject the cells, none showed evidence that their ALS progression was accelerating.

tU-M Medical School ALS Eva Feldman, M.D., Ph.D. is the principal investigator for the trial, and serves as a consultant to NeuralStem as part of her university role. She is also the director of the A. Alfred Taubman Medical Research Institute and the U-M Health System's ALS Clinic.

t"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," says Feldman, who is senior author of the new paper and the Russel N. DeJong Professor of neurology. "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."

tThe trial, which is taking place at Emory University, began in January 2010. 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 FDA has granted approval for the trial to advance to the final two groups of patients (three in each group), all of who will be transplanted in the cervical, or upper, region of the spinal cord.

tNicholas Boulis, M.D., associate professor of neurosurgery at Emory School of Medicine, performs the surgery to implant the cells. He also developed the device used to inject the stem cells into the spinal cord, which received a notice of patent allowance from U.S. Patent and Trademark Office in October.  NeuralStem has purchased an exclusive license to this technology.

tBoulis trained in neurosurgery at U-M, and collaborated on research with Feldman during his seven years of residency. He holds an adjunct associate professor of neurology position at U-M and is one of the Taubman Scholars at the U-M Taubman Institute.

tAlso known as Lou Gehrig's disease, ALS is a fatal neurodegenerative disease with no known cure. It causes the deterioration of specific nerve cells in the brain and spinal cord called motor neurons, which control muscle movement. As the illness progresses, patients lose their ability to walk, talk and breathe. According to the ALS Association, approximately 30,000 Americans have ALS at any given time and patients with the disease usually die within two to five years of diagnosis.

tThis is the first U.S. clinical trial of stem cell injections into the spinal cord for the treatment of ALS. The study is funded by the Maryland-based biotech company NeuralStem, Inc., which is also providing 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, potentially slowing the progression of the disease.

tFor more about the ALS Center and clinical trial at Emory, visit For more about the ALS Clinic at Michigan, visit