Fda Approves Two Drugs For Lou Gehrig’s Disease Patients

After 100 years of offering nothing to amyotrophic lateral sclerosis patients but a death sentence, doctors have two new drugs that show promise for slowing the relentless neuromuscular disease.

The U.S. Food and Drug Administration granted patients this week early access to the drug Myotrophin, an insulinlike growth factor that appears to prevent neuron loss and promote neuron regeneration.

Last year, the FDA approved the drug Rilutek. It, too, seems to slow the disorder, which also is known as Lou Gehrig’s disease for the New York Yankees baseball player who died of it in 1941.

“When I finally got the diagnosis, they told me candidly that there was nothing,” said John Sutton, a Columbus resident who has battled the disease since 1992. “It’s amazing how far things have come in four years.”

Nationally, 6,000 patients are diagnosed with ALS every year. There is no known cause or cure for the disease that usually kills patients three to five years after diagnosis.

The FDA’s early approval of Myotrophin comes after animal tests and four years of human studies.

In the first human trial, 266 ALS patients were randomly assigned Myotrophin or a placebo and were monitored for nine months. Researchers measured motor functions, including swallowing, speaking, standing and walking. Myotrophin had a modest effect in reducing the rate of ALS progression, the FDA said.

“This is the second drug that’s been shown to slow the progression,” said Dr. Jerry Mendell, professor and chairman of neurology at the Ohio State University College of Medicine.

OSU has asked the FDA to designate it as one of the early access dispensing sites for Myotrophin. The drug has not been approved for overthe-counter distribution.

Signs of ALS are progressive weakness and deterioration of the muscles, beginning in the limbs and usually on one side of the body. Inside, the nerves controlling motor function die after their cell bodies become hardened and shriveled.

Death occurs because of respiratory failure - patients simply don’t have the muscles to breath.

One type of the disease damages the motor neurons of the bulbar region of the brain. It weakens the muscles responsible for such vital bodily functions as breathing, swallowing and talking.