The first FDA-approved human stem cell trial to treat amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease will be conducted at the Muscular Dystrophy Association ALS Center at Emory University in Atlanta.
The Food and Drug Administration has given Neuralstem, Inc., a Maryland biotherapeutics company, the go-ahead to conduct a Phase 1 trial to study the safety of Neuralstem’s cells and surgical procedures.
Dr. Jonathan Glass, Director of the Emory MDA/ALS Center will be the site principal investigator.
“This study will be focused on the safety of the procedure for introducing stem cells for the treatment of ALS," he said of the phase 1 trial. “Our hope is to find a surgical approach that is manageable in humans with no negative effects,” said Glass.
“However, as with any phase 1 clinical trial, a patient may benefit from treatment, but that is not the focus of this study,” Glass added.
MDA senior vice president and medical director Dr. Valerie Cwik said “These trials will produce valuable information for us in the ongoing search for effective treatments for ALS. Dr. Glass is a nationally recognized expert in the field of ALS treatment and we are gratified that he will be conducting the human trials at the MDA/ALS Center at Emory University.”
ALS is a disorder that causes paralysis, progresses rapidly and is fatal. There is no treatment or cure for ALS. Once a person is diagnosed, they usually live three to five years. About 30,000 Americans have ALS and approximately 5,000 are diagnosed with the disease every year.
Neuralstem CEO and President Richard Garr stated “While this trial aims to primarily establish safety and feasibility data in treating ALS patients, we also hope to be able to measure a slowing down of the ALS degenerative process.”
The ALS patients will be treated through spinal injections of human neural stem cells. In the first stage of the trial 12 patients will receive five-to-10 stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come about two years later.
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.