Breakthrough could lead to effective treatment for ALS

[boo7382]

Might not be able to help those we currently know with ALS, Alzheimer's, dementia and Parkinson's, but this is still a huge breakthrough. Definitely a step in the right direction.

Researchers say they have found a common cause behind amyotrophic lateral sclerosis, better known as Lou Gehrig's disease, that could lead to an effective treatment.

Dr. Teepu Siddique, a neuroscientist with Northwestern University's Feinberg School of Medicine, said the key was 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 the spinal cord and brain of ALS patients that results in the nervous system slowly losing its ability to carry brain signals to the body's muscular system.

Without those signals, patients gradually are deprived of 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 Monday's issue of the research journal Nature.

ALS afflicts about 30,000 Americans. With no known treatment for the paralysis, 50% 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.

The paper lists 23 contributing scientists, including the lead authors, Northwestern neurological researchers Han-Xiang Deng and Wenjie Chen. Deng and Chen led research that discovered a key protein, ubiquilin 2, in the ALS mystery.

Ubiquilin 2 in spinal and brain system cells is supposed to repair or dispose of other proteins as they become damaged. The researchers discovered a breakdown of this function in ALS patients.

When Ubiquilin 2 is unable to remove or repair damaged proteins, they begin to pile up in the cells, eventually blocking normal transmission of brain signals in the spinal cord and brain, leading to paralysis.

Amelie Gubitz, a research program director at the National Institute of Neurological Disorders and Stroke, said the Northwestern study was a big step forward.

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

http://www.latimes.com/news/nationworld/nation/la-na-als-study-20110822,0,3524095.story

[Carolina Husker]

That's encouraging. ALS is a horrible disease.

[boo7382]

That's encouraging. ALS is a horrible disease.

Yes it is.

Even a small hint to the cause would be a huge breakthrough. As of now, its is a complete mystery. No cause, no treatment, no ability to prevent. You literally just sit around and watch the patient deteriorate on the outside while everything inside is fine. Very very hard to watch.

[dimbee]

A dear friend's father has ALS. It is indeed a horrible disease to witness.

[Hawk]

cool...will it cure my father's parkinson's induced gambling habits?