‘Drug Cocktail’ Doubly Effective
Against ALS in Mice, MDA Scientists Say

Tucson, Ariz., Jan. 22, 2003 — A new study funded by the Muscular Dystrophy Association shows that a combination of two pharmaceuticals — the dietary supplement creatine and the antibiotic minocycline — might make an effective treatment against amyotrophic lateral sclerosis (ALS).

ALS, also known as Lou Gehrig’s disease, is an adult-onset disease that attacks muscle-controlling nerve cells, typically leading to paralysis and death within three to five years of diagnosis. Riluzole (Rilutek), the only drug FDA-approved for treating the disease, extends survival by just a few months.

Robert Friedlander, a professor of neurosurgery at Brigham and Women’s Hospital and Harvard Medical School in Boston, has spent years searching for more potent alternatives to riluzole. In the new study, he shows that mice with ALS given creatine and minocycline develop the disease later and survive longer than mice given either drug alone.

Friedlander’s group reported last year that minocycline extends survival by about 13 percent in mice with ALS. Another team of Harvard scientists had shown in 1999 that creatine has similar effects. Friedlander’s current work, published online today in the Annals of Neurology, reproduces those findings and shows that minocycline and creatine together extend survival of the mice by about 25 percent.

“This is evidence that a combinatorial/cocktail-type approach provides additive protection in the ALS mouse model,” Friedlander said. “This type of approach will likely be what is needed to provide significant protection for humans with ALS.”

The minocycline-creatine combination probably has an additive effect against ALS because each substance appears to target a distinct part of the disease process, Friedlander explained. His previous work showed that minocycline blocks an early event in nerve cell death. Creatine, a natural source of energy in the body, appears to boost energy levels in ailing nerve cells.

MDA is currently funding separate trials of creatine and minocycline in people with ALS.

MDA is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. The Association's programs are funded almost entirely by individual private contributors.