University scientists discover potential ALS treatment from implanted stem cells

By Josh Qian, Daily Staff Reporter
Published April 12, 2012

Hope for patients with the neurological degenerative disease amyotrophic lateral sclerosis, commonly referred to as ALS or Lou Gehrig’s disease, has been found with a recent discovery the University made in partnership with Emory University and Harvard University.

ALS causes nerve cells to stop functioning properly and eventually die. This then leads to muscle weakness, paralysis and eventually death in most ALS patients.

The University’s study — sponsored by NeuralStem Inc., a company with patented technology that produces neural stem cells — was the first to show that stem cells can be safely implanted into the spinal cords of patients with ALS as a form of treatment.

Wiley Online Library, a multidisciplinary collection of online resources, has published the study and Stem Cells, a peer-reviewed science journal, will soon publish the study in print.

Neurology Prof. Eva Feldman — the director of the A. Alfred Taubman Medical Research Institute and the University of Michigan Health System’s ALS Clinic —serves as the principal investigator of the ongoing trials and was the senior author of the paper.

“We have learned so much about stem cells and the promise they hold for effective treatment of devastating diseases like ALS,” Feldman said.

Feldman explained that implanted stem cells wont form the motor neurons that control muscle function and are affected by the disease. Instead, they form a type of support cells, called glial cells, which make contact with motor neurons. The glial cells fill in the gaps of the spinal neurons affected by ALS.

“I like to say these cells clean up the neighborhood that the motor neurons live in,” Feldman said. “These support cells remove toxins, provide nutrition and supply certain proteins that we know help protect the neurons under attack by the disease.”

Feldman said that the team created a special delivery apparatus for implanting stem cells directly into patients’ spinal cords.

“Implanting stem cells in the human spinal cord had never been done before, as far as we know,” she said.

Feldman explained that Nicholas Boulis, neurosurgery professor of Emory and one of Feldman’s past post-doctoral fellows, was a key collaborator in developing the method of implanting the stem cells.

“You have to understand that the spinal cord goes up and down as the patient breathes,” Feldman said. “Boulis devised a way to overcome this obstacle by bolting the delivery device into the plates, or lamina, of the spinal column. It’s really quite a remarkable achievement in itself.”

Feldman added that it took the team years to devise the most effective procedure they first tested on animal models before the U.S. Food and Drug Administration gave the team permission for human clinical trials in January 2010.

“So far 12 individuals have undergone the procedure,” Feldman said. “We have been very encouraged by our ability to safely implant stem cells in the human spinal cord, but we have a long way to go.”

Feldman said the patients who have gone through the procedure have not experienced any serious adverse side effects, and some patients have shown a reduction in the progression of the disease.

“We do extensive tests on the patients before they leave the hospital and follow them for months,” Feldman said. “All of our findings point to the safety of the procedure.”

Though Feldman said it is too early to evaluate the efficacy of the treatment, she believes the FDA will approve it for widespread clinical use in three to four years. She added that she expects multiple breakthroughs in stem cell research application in the coming years.

“We are making great progress in laboratories around the world … I think we will be amazed 20 years from now what we will be able to do with stem cell technology.”