Research Findings

TUCSON, Ariz., Oct. 31, 2002 - The Muscular Dystrophy Association and the National Institutes of Health are supporting a vastly expanded, 400-person, multicenter study of the drug minocycline in amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease). A recently completed study of about 40 people showed promising results.

Neurologists Paul Gordon, who heads the MDA/ALS Center at the University of New Mexico Health Sciences Center in Albuquerque, and Robert Miller, director of the MDA/ALS Center at California Pacific Medical Center in San Francisco, will direct the trial at 20 centers in the United States and one in Canada.

The study is likely to begin recruiting participants in early 2003.
MDA Director of Research Development Sharon Hesterlee said, "This study is exciting because minocycline is a well-known drug that could be offered to patients immediately, if it proves effective."

Minocycline, an antibiotic in the tetracycline family, is approved by the U.S. Food and Drug Administration for treatment of infections. The drug has recently been shown to interfere with a type of cell death that may play a major role in ALS.

ALS affects both sexes, usually beginning in middle age, and destroys muscle-controlling nerve cells in the brain and spinal cord. The loss of nerve cells leads to progressive paralysis and usually to death three to five years after onset. No cure exists.

In addition to its infection-fighting properties, minocycline appears to block the release of a chemical called cytochrome c from inside the cell. When cytochrome c is released from the cell's energy-producing subunits (the mitochondria), a chain of events resulting in cell death apparently results.

A drug that blocks cytochrome c release might block the cell death pathway and save nerve cells in ALS and other degenerative diseases, researchers say.

Earlier this year, Gordon and Miller found that minocycline was tolerated well by the majority of some 40 people with ALS who took it in high doses in a pilot study. Nevertheless, Gordon cautions against taking minocycline for ALS outside a formal study, where serious side effects can be monitored.

The pilot study wasn't designed to test effectiveness of the drug against ALS, he emphasized, but the new, larger study will be.

"We're quite excited," Gordon said. "We think minocycline has a unique mechanism of action as both an antiapoptotic [anti-cell death] and an anti- inflammatory agent. Medications like this haven't been tested before in ALS, so we're hopeful."

MDA is a voluntary health agency working to defeat more than 40 neuromuscular diseases. The Association has been vitally active in ALS research and services for more than 50 years, and has invested more than $135 million in its ALS program to date. It supports 29 MDA/ALS research and clinical centers across the country.