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Credit: MD Anderson Cancer Center

Three simultaneous safety and efficacy studies of the drug larotrectinib reported an overall response rate of 75 percent for patients ages four months to 76 years with 17 different cancer diagnoses. All patients had tumors with tropomyosin receptor kinase (TRK) fusions, gene mutations that switch on TRK genes, allowing cancer growth. The studies indicate larotrectinib as a potentially powerful new treatment approach for the approximately 5,000 patients with these forms of cancer.

Findings from the multi-center investigation were published in the Feb. 21 online issue of the New England Journal of Medicine (NEJM). Co-principal investigators were David Hong, M.D., professor of Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center, and David Hyman, M.D., chief of Early Drug Development at Memorial Sloan Kettering Cancer Center.

“In this series of studies, larotrectinib had rapid, potent and durable anti-tumor activity in children and adults who had solid tumors with TRK fusions without regard to patient age, tumor tissue and fusion status,” said Hong. “Our data not only validated TRK fusions as therapeutic targets, but also showed the potential for larotrectinib as a therapeutic agent for

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