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 stated that, after reviewing safety data from the first 12 patients, the US Food and Drug Administration (US FDA) has granted approval for the trial to advance to transplanting 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.
“The goal of our cell therapy programme is to create therapies that will slow down, stabilize, or reverse, functional deficits in central nervous system (CNS) diseases,” said Karl Johe, PhD, Neuralstem Chairman and Chief Scientific Officer. “By moving the cell delivery to cervical spinal cord--the first time the US FDA has approved intraspinal injections in this region--we will demonstrate that we can deliver our cells safely and routinely to all parts of the spinal cord.”
The trial is under the direction of Principal Investigator, Eva Feldman, MD, PhD, 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, MD, Director of the Emory ALS Centre. The surgeries are performed by Emory Neurosurgeon, Nicholas M. Boulis, MD. 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.”
“Prolongation of life for patients with ALS will require therapeutic intervention at the level of the cervical spinal motor neurons affecting respiratory function,” they 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.”
“We are very excited about the trial results to date and encouraged by the safety data,” commented Lucie Bruijn, PhD, Chief Scientist, The ALS Association. “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.”
“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.”
The phase I trial to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in ALS patients has been underway since January 2010. The trial is designed to enroll up to 18 patients. All of the first 12 patients have been transplanted in the lumbar (lower back) region of the spine. The trial will now progress to the last six patients. The first three of these will receive unilateral injections in the cervical region of the spine. The next three will receive bilateral injections in the cervical region.
Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia.