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Existing Drug May Lead to ALS Treatment

Researchers hope to start testing in patients within five years

TUESDAY, Oct. 20, 2009 (HealthDay News) -- A drug similar to one now used to treat blood poisoning holds promise as a treatment for amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig's disease, researchers say.

At the moment, the drug has only been tested in mice, and there's no guarantee that it would have similar effects in humans, the scientists noted.

In the study, the researchers were able to extend the life span of mice with a severe form of the disease by 25 percent. The mice were also better able to live normal lives and didn't suffer from muscle degeneration as quickly.

ALS, which harms the motor-related neurons in the brain, brainstem and spinal cord, causes paralysis and usually leads to death within five years. There is currently no cure or treatment that can slow progression of the disease.

The new research found that a compound already used in other patients appears to protect neurons that are under attack in ALS. The compound, a form of an enzyme known as activated protein C, is already used to treat blood poisoning, but it can cause an increased risk of bleeding, and researchers don't think it will work as a treatment for ALS. As a result, they are looking for ways to tinker with the compound to boost its chances of serving as an appropriate treatment.

Study co-author Dr. Berislav Zlokovic, a neuroscientist at the University of Rochester Medical Center, noted in a university news release that there is one benefit to the compound: It's approved for use through injection, while other treatments under development need surgery to be administered.

The researchers hope to test the treatment in humans within the next five years.

The research was published online Oct. 19 in the Journal of Clinical Investigation.

More information

Learn more about amyotrophic lateral sclerosis from the ALS Association.

SOURCE: University of Rochester Medical Center, news release, Oct. 19, 2009
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