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New Hope for Lou: Unexplored Therapeutic Targets for ALS

Savannah, Ga. (September 3, 2015)—Amyotrophic lateral sclerosis (ALS) is a disorder in which cells of the nervous system die, leading to muscle weakness that impacts breathing, movement and other physical functions. More commonly known as Lou Gehrig’s disease, ALS has no cure, and the only approved therapy slows the progression by only a few months. In a new study to be presented at the 14th International Conference on Endothelin: Physiology, Pathophysiology and Therapeutics, researchers from Johns Hopkins University have identified an unexplored avenue of treatment for ALS.

Endothelin (ET)-1, a small protein produced by blood vessel cells and a powerful vessel constrictor, is also produced by astrocytes, cells in the brain that studies are revealing play many roles in health and disease. ALS progression is associated with the dysfunction of astrocytes, and earlier studies have shown that ET-1 influences a number of cellular pathways implicated in ALS progression. Gene expression studies also suggest that levels of ET-1 and the receptor it binds to, ET-B, are elevated in patients with ALS.

This new study investigated whether levels of ET and its receptor, ET-B, were altered in the regions where nerve cells die in ALS. The researchers examined ALS patient-derived tissue samples and cells and a mouse model of ALS using a range of gene expression and protein measurement techniques. They found a higher level of ET-1 in astrocytes and a higher level of the ET-B receptor in nerve cells in regions affected in ALS. They also found variations in the gene sequences for ET-1 in patients with an inherited form of ALS.

“These experiments demonstrate striking abnormalities in the central nervous system endothelin system, [suggesting that] the endothelin system may represent a largely unexplored and potentially significant target for therapeutic intervention in ALS,” according to the researchers.

Lyle Ostrow, MD, PhD, assistant professor of neurology and pathology at Johns Hopkins University, will present “The Endothelin System in Amyotrophic Lateral Sclerosis (ALS)” at the poster session on Thursday, September 3, from 1 to 2:30 PM EDT in Ballroom BCDEF and as part of the symposium “Central Nervous System” on Saturday, September 5, at 9 AM EDT in Ballroom A of the Hyatt Regency Savannah.

NOTE TO JOURNALISTS: To read the full abstract or to schedule an interview with a member of the research team, please contact the APS Communications Office or 301-634-7209. Find more research highlights in the APS Press Room.

Physiology is the study of how molecules, cells, tissues and organs function in health and disease. Established in 1887, the American Physiological Society (APS) was the first U.S. society in the biomedical sciences field. The Society represents more than 11,000 members and publishes 14 peer-reviewed journals with a worldwide readership.



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