ALS Drugs Have New Target Thanks to a Protein Interaction

January 29, 2008 at 3:24 PM Drug News
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ALS Drugs Have New Target Thanks to a Protein Interaction
A study conducted by researchers at the University of Iowa has resulted in a discovery of a protein-protein interaction that has lead to the identification of a drug that slows the progression of amyotrophic lateral sclerosis in mice and nearly doubles the animals' lifespan. The study is published in the Jan 24 online issue of the Journal of Clinical Investigation.

The UI findings may lead to a treatment for some forms of ALS, and the research also reveals a biological mechanism that might represent a new drug target for ALS and other neurological diseases.

ALS, also known as Lou Gehrig's disease, is a fatal, progressive neurodegenerative disease that affects the motor nerve cells of the brain and spinal cord. Degeneration of motor neurons impairs muscle control and movement and eventually leads to paralysis and death. "Sporadic" ALS, which can affect anyone, is the most common form of disease accounting for 90 to 95 percent of all case in the United States. About 5 to 10 percent of ALS cases are inherited.

While studying the basic biology of cell signaling, scientists led by John Engelhardt, Ph.D., professor and head of anatomy and cell biology in the UI Roy J. and Lucille A. Carver College of Medicine, made the unexpected discovery that superoxide dismutase-1 (SOD1), a protein that is mutated in inherited forms of ALS, interacts with Rac1, a protein that regulates the production of reactive oxygen species (ROS) by the Nox2 protein complex.

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