U-M to lead stem cell trial for Lou Gehrig's disease treatment

Apr 17, 2013

Credit University of Michigan

The University of Michigan is set to lead a national trial in the use of stem cell injections to study their effects on the symptoms of ALS, or Lou Gehrig's disease.

U-M will collaborate with Emory University in the Phase II clinical trial, pending approval by  the Institutional Review Board, which could take about a month. The FDA has approved the trial.

It's estimated between 30,000 to 50,000 people in the United States have ALS.

"It's a disease of the large motor nerve cells in the spinal cord and brain, and when they become diseased, the nerve that connects to the muscle becomes diseased, and the muscle becomes weak," explains U-M Professor of Neurology Dr. Eva Feldman, who has studied ALS for 25 years and is directing the study.

"Over time, people experience a progression of weakness of their arms and legs and muscles around the face -- the muscles that control swallowing and breathing. We're going to take neuro stem cells and transplant them into the upper part of the spinal cords of people with ALS," she says. "The goal is to show safety, and then to begin to see if it offers some therapeutic efficacy in terms of allowing people to breathe longer."

She says the study will also examine whether the injections help preserve a patient's strength, and whether there's a change in the patient's quality of life.

Feldman says Michigan has a relatively high incidence of the disease, which researchers believe may be related to environmental causes.

She says 80 to 85 percent of ALS cases are sporadic, but up to 15 percent appear to have a hereditary factor in their disease.

"If it runs in your family, if you have a parent or sibling with ALS, you have a 50 percent risk" of developing the disease, Feldman says.

"The average patient is between 50 and 60 years old, and it's a male-predominant disorder, which means they're more likely to get it than women," she explains. "And from diagnosis to death is somewhere between three to four years in the average patient, so it can be a very aggressive disorder."

Patients can learn more about this and other studies by visiting clinicaltrials.gov.