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$4.37 million NCI grant accelerates recent laboratory finding in Ewing's sarcoma
Date:10/18/2010

Washington, DC The National Cancer Institute has awarded a $4.37 million grant to Jeffrey A. Toretsky, MD, at Georgetown Lombardi Comprehensive Cancer Center, to advance a recent scientific laboratory discovery into a new treatment for Ewing's sarcoma. The grant, issued under the American Recovery and Reinvestment Act, will help fund preclinical toxicology studies of a new agent identified by Toretsky and his team a critical step in drug development that usually is stalled for years because of cost.

"A significant hindrance of therapeutic development is translation from the laboratory to the clinic," says Toretsky. "This funding not only allows this step to occur, but it could dramatically accelerate the time of taking our laboratory finding and turning it into a therapy for patients."

If preclinical toxicology studies are successful, a clinical trial with the agent would follow. The studies of the agent are based on a 2009 discovery in which Toretsky and his colleagues found a novel way to block the activity of the fusion protein responsible for Ewing's sarcoma, a rare cancer found in children and young adults. Toretsky described the finding in the journal Nature Medicine.

He and his colleagues discovered and successfully tested a small molecule that keeps the fusion protein from sticking to another protein that is critical for tumor formation. The researchers say this interaction is unique and is especially surprising since the Ewing's sarcoma fusion protein is extremely flexible, which allows it to change shape constantly.

"Most targeted small molecule cancer drugs inhibit the intrinsic activity of a single protein, but our agent stops two proteins from interacting. This has never been shown before with a cancer-causing fusion protein and represents a potentially novel medical therapy," says Toretsky.

Agents currently in use that work against fusion proteins inhibit a single protein to stop intrin
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Contact: Karen Mallet
km463@georgetown.edu
215-514-9751
Georgetown University Medical Center
Source:Eurekalert

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