A new study by researchers at the University of Pittsburgh School of Medicine may hold promise for the understanding and treatment of amyotrophic lateral sclerosis, the neurodegenerative disease more commonly known as ALS or Lou Gehrig's disease.
The study, published by the journal Neurobiology of Disease, showed that when mice received injections of melatonin, a hormone, they developed ALS symptoms later and lived longer than mice who were given a placebo.
It's too soon to say whether the results seen in mice will translate into results for humans with the disease, said Robert Friedlander, the study's senior investigator and chairman of Pitt's Neurological Surgery department. It will be "a number of years" before human trials can offer any insight, he said.
ALS, which affects about 30,000 Americans at any one time, is a disease that causes the motor neurons to degenerate. A person with ALS loses the ability to control muscle movement, then experiences paralysis and trouble swallowing and breathing. The average life expectancy for a person with ALS is two to five years after diagnosis.
Currently, the only drug treatment for ALS is Rilutek, though it only extends a person's lifetime by a few months. Dr. Friedlander and his researchers screened 1,040 FDA-approved drugs before discovering that melatonin could prevent cell death in neurons.
Although melatonin is currently available in stores, Dr. Friedlander cautioned that more research remains to determine if the drug could slow the progression of ALS in humans. He predicted that the eventual treatment for ALS will be a "cocktail-type approach," involving multiple drugs, similar to HIV treatment.
Still, Dr. Friedlander said the study's results -- and the insight they provide into the pathways affected by ALS -- are a good step forward, especially since little progress has been made since New York Yankees great Gehrig died in 1941.
"There is increased knowledge that we're gaining every day," he said. He added, "I don't want to say we're at the edge of a breakthrough." But he said the more drugs researchers are able to show work in mice and then move them into human trials, the closer researchers are to arriving at treatments.
"We are on the right path, and we wish we could get there faster."