Neuralstem Receives FDA Approval to Dose Patients in Cervical Region in Ongoing ALS Safety Trial
Neuralstem, Inc. has updated the progress of its ongoing Phase I safety trial of the company’s spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) at Emory University in Atlanta, Georgia. The company announced that, after reviewing safety data from the first 12 patients, the Food and Drug Administration (FDA) has granted approval for the trial to advance to transplanting cells into patients in the cervical (upper back) region. Until now, patients have received injections in the lumbar (lower back) region only. Earlier this summer, the trial’s Safety Monitoring Board unanimously approved moving to the cervical injection phase. The trial will now advance to the final two cohorts of patients with ALS, all of whom will be transplanted in the cervical region of the spine.
Cells injected into the cervical region have the possibility of supporting breathing function. The cells injected in the lumbar region, as with the first 12 patients, cannot impact breathing.
“This is a key milestone in the progression of the trial,” said Karl Johe, Ph.D., Neuralstem Chairman and Chief Scientific Officer. “We need to demonstrate that we can deliver the cells safely and routinely throughout all regions of the spinal cord.”
The trial is under the direction of Principal Investigator, Eva Feldman, M.D., Ph.D., Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System, and Jonathan D. Glass, M.D., Director of the Emory ALS Center. The surgeries are performed by Emory Neurosurgeon, Nicholas M. Boulis, M.D. Dr. Feldman is an unpaid consultant to Neuralstem.
In a joint statement, the three doctors concluded: “We have successfully treated 12 ALS patients with unilateral or bilateral intraspinal injections of neural stem cells. All of the patients tolerated the procedure without major surgical complications, and there are no indications to date that the stem cells themselves are either toxic or injurious to the spinal cord. Our quantitative clinical assessments showed no evidence of acceleration of disease following stem cell injections, meeting our stated goal of proving safety for this Phase I trial. We have cautious optimism that a few of the patients may have slowed in their progression of lower extremity weakness, and one patient may have improved.”
“We are very excited about the trial results to date and encouraged by the safety data,” commented ALS Association Chief Scientist Lucie Bruijn, Ph.D. “This is a very important step forward for stem cell therapy in ALS, and we are fortunate to have such a committed and skilled research team at Emory and Neuralstem to move this forward. These results pave the way for the next stage of the trial and for future stem cell transplants in ALS.”
Prolonging life for people with ALS will require therapeutic intervention at the level of the cervical spinal motor neurons affecting respiratory function,” the three doctors continued. “To reach this ultimate goal, we plan to move to injections into the cervical spinal cord, which is the next stage of this Phase I trial.”
“This is a major milestone for Neuralstem,” said Richard Garr, Neuralstem CEO and President. “Demonstrating the feasibility and safety of transplanting our cells in the upper regions of the spinal cord will have important ramifications for our spinal cord injury program as well as ALS.”