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.

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

All 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.

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

“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 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.”

First author Dr. Jonathan Glass, director of Emory’s ALS Center, notes that the results have already led to a next phase of the trial at Emory. “For these first 12 patients, we have met the objective of the Phase I trial, demonstrating safety for both the procedure of intraspinal injection and the presence of the neural stem cells in the spinal cords of ALS patients,” he says. “We are encouraged by these results and have now advanced our trial to injections into the cervical spinal cord, targeting the motor neurons that control respiratory function.” Glass is a professor of neurology and pathology at Emory University School of Medicine. The Emory trial is only open to patients who live near Atlanta.

The trial 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.

Dr. Nicholas Boulis, 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.

Boulis 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 Taubman Institute.

“We hope this trial continues to foster the development of both new techniques for surgical implementation and treatments for people who are living with ALS,” Boulis says. “We are still early in this trial; however, these initial results allow us to move forward with optimism.”