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NIH Record - National Institutes of Health

Researchers Make Advance in Possible Treatments for Gaucher, Parkinson’s Diseases

With assistance from high-throughput drug screening, NIH researchers have identified and tested a molecule that shows promise as a possible treatment for the rare Gaucher disease and the more common Parkinson’s disease. Dr. Ellen Sidransky, a senior investigator with NHGRI, and her collaborators at NINDS and NCATS published their findings June 12 in the Journal of Neuroscience.

“Until now, drugs used to treat Gaucher disease have not been able to enter the brain and reach those neurons that are affected in the most severe forms of Gaucher disease or in Parkinson’s disease,” said Sidransky. “It’s really exciting to have found a molecule that theoretically could be widely available to treat people with these diseases. However, there’s a long distance between identifying this molecule and having an approved drug.”

Sidransky has conducted research on Gaucher disease for the last 28 years and made the connection between Gaucher disease and Parkinson’s disease in 2001.

“This research constitutes a major advance,” said Dr. Daniel Kastner, NHGRI scientific director. “It demonstrates how insights from a rare disorder such as Gaucher disease can have direct relevance to the treatment of common disorders like Parkinson’s disease.”

Researchers will next test the new molecule to see if it might be developed into an appropriate prototype drug for patients with Gaucher disease and Parkinson’s disease.

Gaucher disease affects an estimated 1 in 50,000 to 1 in 100,000 people in the general population. People of Eastern and Central European (Ashkenazi) Jewish heritage are more likely to get Gaucher disease. Parkinson’s disease affects 1.5-2 percent of people over age 60, and the incidence increases with age. In the United States, about 60,000 new cases are identified each year. Parkinson’s disease affects more than 1 million people in North America and 7 million-10 million people worldwide.

The NIH Record

The NIH Record, founded in 1949, is the biweekly newsletter for employees of the National Institutes of Health.

Published 25 times each year, it comes out on payday Fridays.

Associate Editor: Carla Garnett
Carla.Garnett@nih.gov

Staff Writers:

Eric Bock
Eric.Bock@nih.gov

Dana Talesnik
Dana.Talesnik@nih.gov

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