Experimental Drugs for Lou Gehrig's Disease

Lou Gehrig lived for two years after being diagnosed in 1939 with amyotrophic lateral sclerosis (ALS), the disease that now bears his name. For decades, little changed. Most ALS patients die within three to five years after they first have
symptoms, according to the ALS Association. A May 29, 2008, article on the ALS website alsa.org said "patients with Lou Gehrig's disease face a dismal prognosis. The only approved drug, Sanofi-Aventis' Rilutek, slows the fatal muscle-wasting disease by just a few months." In the last year, though, at least five experimental drugs have been tested on ALS patients.

Potential Breakthrough

• Spinal cord stem cells produced by Neuralstem, Inc. are being tested as treatment. "In work with animals, these spinal cord stem cells both protected at-risk motor neurons and made connections to the neurons controlling muscles," Dr. Eva Feldman, the director of the University of Michigan Health System ALS Clinic, told the ALS Association. "We don't want to raise expectations unduly, but we believe these stem cells could produce similar results in patients with ALS."

Optimism

• The ALS Association is so optimistic that lithium will slow the progress of early stage ALS that it is funding a major clinical trial of the drug.

Working in Rats

• Tests to treat a familial form of Lou Gehrig's disease with antisense therapy are underway. This new therapy consists of trying to shut down the RNA (ribonucleic acid) that produces disease-causing proteins.

Targeting Mutations

• Arimoclomol is being tested on people with "rapidly progressive forms" of familial Lou Gehrig's disease, according to a March 11, 2009, news release by the ALS Association. It targets ALS caused by mutations in the superoxide dismutase (SOD1) gene.

Bad News

• The news isn't all good. One experimental drug, myotrophin, has already failed, Lucie Bruijn, the ALS Association's senior vice president for research and development, reported.