Drug Slows Progression Of Lou Gehrig's Disease

Results of a nine-month study involving 266 patients at eight North American medical centers were reported in the December issue of the journal Neurology.

"Patients taking a high dose of the medication progressed 26 percent slower than patients on the placebo or inactive drug," said Dr. Eugene Lai, a neurologist at Baylor College of Medicine in Houston and lead author of the journal article. "They also experienced a slower decline in quality of life."

Lou Gehrig's disease, also called amyotrophic lateral sclerosis or ALS, involves the progressive breakdown of motor neurons, the nerve cells that control muscular activity. It causes severe muscle weakness, difficulty in speaking, swallowing and breathing, and ultimately death.

"To ALS patients, slowing progression can mean maintaining arm and leg strength longer, delaying the onset of speech problems and prolonging mobility and independence," said Dr. Stanley Appel, director of the MDA/ALS Clinic at Baylor and The Methodist Hospital and Baylor chairman of neurology.

The study drug, Myotrophin or recombinant human insulin-like growth factor-I (rhIGF-I), is a man-made form of the natural protein insulin-like growth factor-I. This protein is important for normal human growth and development.

"The growth factor may act in a variety of ways on the motor neuron, nerve and skeletal muscle. It has the ability to induce nerve sprouting and growth and to promote nerve cell survival," Appel said. "These actions are important to ALS patients."

The medication was given twice daily as a shallow injection under the skin, and participants received either a low dose, a high dose or a placebo.

Patients were evaluated monthly using the Appel ALS rating scale to assess speech and swallowing, respiratory function, arm and leg muscle strength, and arm and leg function. The Sickness Impact Profile measured participants' perceptions of their quality of life, including psychological well-being, daily living and disability.

"The drug proved to be well tolerated and simple for patients to administer," Lai said.

Treatment-related side effects were relatively mild and included injection-site inflammation, hair change, knee pain and facial swelling. Less than five percent of the patients left the study due to the side effects.

Myotrophin currently is under review and pending marketing approval by the Food and Drug Administration.