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Protein May Slow Progression of Lou Gehrig’s Disease

Last Updated: October 26, 2009.

Activated protein C slows disease progression and improves survival in a mouse model of amyotrophic lateral sclerosis, according to a study published online Oct. 19 in the Journal of Clinical Investigation.

MONDAY, Oct. 26 (HealthDay News) -- Activated protein C slows disease progression and improves survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to a study published online Oct. 19 in the Journal of Clinical Investigation.

Zhihui Zhong, Ph.D., from the University of Rochester Medical Center in New York, and colleagues used mice containing a mutation in the superoxide dismutase-1 (SOD1) gene linked to ALS as a model of the disease to study the effect of administering activated protein C, a protease with anticoagulant activity.

The researchers found that active activated protein C was able to cross the blood-spinal cord barrier, whose integrity is impaired early in the disease process. This was associated with elimination of leakage of potentially neurotoxic hemoglobin-derived products across the blood-spinal cord barrier. Activated protein C reduced the expression of SOD1 in microvessels, motor neurons, and microglial cells, and slowed disease progression and extended survival when given after disease onset.

"With the recognition that accumulation of aberrant SOD1 species has been linked to most cases of sporadic ALS, strategies based on activation of the protein C cellular pathway are promising directions for treating patients with familial, and possibly sporadic, ALS," Zhong and colleagues conclude.

One author is a founder of ZZ Biotech, which is focused on treating various neurological problems, while two authors have issued and pending patents related to activated protein C.

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