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Discovery may yield treatment for ALS

Researchers say they found a common cause behind amyotrophic lateral sclerosis that could lead to an effective treatment for the mysterious and deadly affliction better known as Lou Gehrig's disease.

Dr. Teepu Siddique, a neuroscientist with Northwestern University's Feinberg School of Medicine whose pioneering work fueled the research team's work, said the key to the breakthrough lies in the discovery of an underlying disease process for all types of ALS.

The discovery also could help in developing treatments for other, more common neurodegenerative diseases such as Alzheimer's, dementia and Parkinson's, Siddique said.

The Northwestern team identified the breakdown of cellular recycling systems in the neurons of ALS patients' spinal cords and brains that results in the nervous system slowly losing its ability to carry signals to the body's muscular system.

Without those signals, patients gradually lose the ability to move, talk, swallow and breathe.

"This is the first time we could connect [ALS] to a clear-cut biomedical mechanism," Siddique said. "It has really made the direction we have to take very clear and sharp. We can now test for drugs that would regulate this protein pathway or optimize it, so it functions as it should in a normal state."

The announcement of the breakthrough appears in today's issue of the research journal Nature. The paper lists 23 contributing scientists, including the lead authors, Northwestern neurological researchers Han-Xiang Deng and Wenjie Chen.

ALS afflicts about 30,000 Americans. With no known treatment for the paralysis, 50 percent of all ALS patients die within three years.

In 1941, New York Yankee baseball star Lou Gehrig died at 37 of the disease that now carries his name.

Amelie Gubitz, a research program director at the National Institute of Neurological Disorders and Stroke, said the Northwestern research is a big step forward in worldwide efforts to conquer ALS.

"You need to understand at the cellular level what is going wrong, then you can begin to design drugs," Gubitz said.

"ALS is a complicated problem, and Dr. Siddique's research adds a big piece to the puzzle that gives us important new insights."

ALS has three forms: "familial," which is hereditary and passed through genes; non-hereditary, which is called "sporadic"; and "ALS/dementia," which targets the brain.

Siddique was part of a study that made a major breakthrough in the early 1990s, discovering the "familial" gene that transmits the disease within some families. That followed the launch 25 years ago of an ongoing study of an East Coast family that has lost more than 20 members to ALS.

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