Blood biomarker for ALS points to new therapy

Researchers from Brigham and Women's Hospital have found a blood biomarker that could detect amyotrophic lateral sclerosis (ALS), a progressive degenerative neurological disease that affects about 30,000 Americans. The disease is also known as Lou Gehrig's disease, after the New York Yankees baseball player who died from the condition. Other notable people who have died from or are living with ALS include professor Stephen Hawking, actor David Niven, musician Jason Becker and Chinese leader Mao Zedong.

The researchers looked at mice with a genetic mutation triggering a disease that mimics ALS, and found that monocytes, a type of immune cell involved in inflammation, were activated about two months before the onset of the disease, and then as the disease started to take hold, flooded to the spinal cord, and this influx was linked with nerve cell death.

"When we discovered the monocytes were coming into the brain and causing damage in the animals, we asked, 'If we can affect those cells and change them--if we could decrease their inflammatory nature, could the animal do better?'" Howard Weiner, director of the Brigham and Women's Hospital Multiple Sclerosis Program, said to FoxNews.  "We're reluctant to use the word cure, but it could slow down the disease and maybe stop it from progressing."

By treating the mice with antibodies that blocked this aberrant and inflammatory immune response, fewer monocytes entered the spinal cord region, fewer nerve cells died, and the mice lived longer. The researchers looked at blood samples from people with ALS, and found similar levels of monocytes and similar inflammatory responses, suggesting that the findings in mice could be applied to humans. The research could lead to a simple blood test to diagnose the disease and track its progression, as well as point the way toward new treatments--currently Rilutek (riluzole) from Sanofi-Aventis ($SNY) is the only approved drug for this devastating disorder.

"We now have a blood test where we can test ALS patients for this inflammatory signature of monocytes in the blood. And we have a new target therapy to target these monocytes and stop them from going in the spinal cord," said Weiner, who predicts clinical trials within three years.

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