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ITHS Member receives NIH Funding

ITHS Member receives NIH Funding

froehnerIn the world of drug development, researchers may experience many false starts and tests before a compound successfully makes it through clinical trials and enters the market.  In fact, approximately 1 in 12 treatments ever make it to patients. What happens to promising molecular compounds that have undergone extensive pre-clinical and safety testing but don’t quite reach the market?

The NIH created a pilot program called Discovering New Therapeutic Uses for Existing Molecules to determine if compounds can be repurposed. Private companies provide researchers with their compounds and related data while the NIH NCATS program provides funding through a cooperative agreement research grant.

Stanley C. Froehner, Professor and Chair, Department of Physiology & Biophysics at the University of Washington and Dr. Kathryn R. Wagner of Kennedy Krieger Institute are recent recipients of a $2.3 million grant for the first year of an anticipated 3 year grant to test a drug that may address Duchenne muscular dystrophy (DMD). As a disabling and fatal disorder that affects approximately 1 in 5,000 male births worldwide, DMD has very limited treatment options.

Froehner and Wagner will test Sanofi’s investigational drug in mouse models to determine if it improves skeletal muscle function. “This collaboration with Sanofi to test their drug is possible only because of this new NIH program,” says Froehner. After his team runs the toxicology studies, they will work with Wagner’s team on the downstream clinical trials in human patients.

The ITHS helped Froehner in a number of ways to prepare for the funding. He first received a letter of support that outlined the type of services available. Then ITHS connected Froehner to experts in study design to help determine specifics for a toxicology study and drug delivery in food.

“I had no idea ITHS provided all this expertise,” said Froehner. “To have all these people supporting me has been a huge help.”