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'U' researcher's landmark stem cell trial gets FDA green light

BY VALIANT LOWITZ
Daily Staff Reporter
Published September 22, 2009

Correction appended: An earlier version of this article incorrectly referred to the study as the first clinical trial of a stem cell-derived treatment in a reference toward the bottom of the article without clarifying that it was the first of its kind for ALS, not the first of its kind generally.

In a major victory for proponents of stem cell research, the U.S. Food and Drug Administration approved the first-ever human clinical trial of a stem cell treatment for Lou Gehrig’s Disease last Friday. The study will be led by a University of Michigan expert in the field.

Dr. Eva Feldman, the director of both the U-M Amyotrophic Lateral Sclerosis (ALS) Clinic and the Program for Neurology Research and Discovery, helped develop the treatment and will act as principal investigator for the landmark trial.

Feldman worked with a team of neurologists to develop the new protocol for injecting stem cells into the spinal chord of patients. This system, researchers hope, will reconnect severed lines of communication between the cells of paralyzed ALS patients and delay or reverse some of the disease’s effects.

In an interview yesterday, Feldman discussed the study and what lies ahead for her team of researchers.

ALS, commonly known as Lou Gehrig’s disease, is a progressive neural degenerative disorder that affects roughly 30,000 Americans, according to the ALS Association. About 5,600 people in this country are diagnosed with the disease every year — or 15 new cases every day.

The disease affects nerve cells in the brain leading to the degeneration of motor neurons in the spinal chord. The patient eventually loses control of all voluntary muscle movement. Cognitive functions are usually left undamaged. Notable figures suffering from ALS include renowned physicist Stephen Hawking and musician Jason Becker.

“Currently ALS is a fatal neurodegenerative disease,” said Feldman. “The only available treatment is medication given by mouth.”

Though these medications slow the progression of the disease, Feldman said, they may only increase survival by around three to five months.

The clinical treatment injects stem cells directly into the spinal cord, protecting at-risk motor neurons and potentially rebuilding severed connections.

“We very much want to find a way to stop the progression of ALS,” Feldman said, “rather than just slow it down.”

The Investigational New Drug application was submitted to the FDA by Neuralstem Inc., a Maryland-based biotherapeutics company that is sponsoring the trial and providing all of the neural stem cells used in the treatments.