Pitt findings on Lou Gehrig's disease may hold hope

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A person diagnosed this year with amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig's disease, will receive basically the same prognosis as the New York Yankee did -- an average life expectancy of two to five years.

More than 70 years after Gehrig's death, the cause of the disease that took his life and then his name remains unclear, and with the exception of one federally approved drug that has been shown to extend life a few months, there is no effective treatment and no cure. Although research into ALS continues, studies in recent years have been marked by "frustrating failures," said Lucie Bruijn, chief scientist for the ALS Association.

But researchers at the University of Pittsburgh School of Medicine announced Thursday the results of a new study on ALS symptoms in mice that they hope may hold promise for the understanding and treatment of the disease.

The study results, published by the journal Neurobiology of Disease, showed that when mice received injections of the hormone melatonin, 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. And it will be a number of years before human trials can offer any insight, he said.

In ALS research, that question -- whether what works in mice can work in humans -- is the big one.

"That's the challenge in our field, really," said Ms. Bruijn, who has a doctorate in biochemistry. "The challenge for us is to move from the mouse model and findings in the mouse model and be effective in the clinic."

She said she was enthusiastic but cautious about melatonin as an ALS treatment, citing an earlier study that demonstrated the compound's antioxidative effects on mice.

Dr. Friedlander, who also stressed the importance of waiting to see whether something that works in mice could work in humans, described the study's results as a good step forward.

"There is increased knowledge that we're gaining every day," he said. "I don't want to say we're at the edge of a breakthrough."

But he said the more drugs that 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," said Dr. Friedlander, who has been researching ALS for 20 years. This study began five years ago.

Time is of the essence for people with ALS, which affects about 30,000 Americans, including 300 in Western Pennsylvania, at any one time. The disease leaves the mind intact while it 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, causing death two to five years after diagnosis on average.

Rilutek, or riluzole, is the sole FDA-approved drug treatment available for ALS, and it extends a person's lifetime by a few months. Melatonin emerged as a possible treatment for ALS-like symptoms in mice after Dr. Friedlander and his researchers screened 1,040 FDA-approved drugs, discovering that melatonin was one of the most effective compounds in preventing neuron death.

In mice, it delayed ALS symptoms, neurological deterioration and death by 7 percent of the mouse's lifetime, the study showed. The importance of the study, Dr. Friedlander said, is that it identified a new potential treatment for ALS and a pathway for the disease that may help researchers better understand it.

Although melatonin is available in stores, Dr. Friedlander said he would not endorse self-treatment and cautioned that further research trials must be done. He also predicted that the eventual treatment for ALS will be a "cocktail-type approach," involving multiple drugs, similar to HIV treatment.

"That's what we need in a lot of these neurodegenerative diseases, including ALS, is to identify a group of drugs that will act in concert and synergistically to have an effect," he said.

ALS is a difficult disease, Ms. Bruijn said, and many times a treatment has looked promising but didn't work in patients. Still, she predicted news of the Pitt study would be greeted with optimism by people with ALS.

"There will be great excitement among patients, because there's a desperation for treatment," she said

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Kaitlynn Riely: kriely@post-gazette.com or 412-263-1707.


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