Clinical trial studies new therapy in children and adults with neurofibromatosis-related GIST

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Gastrointestinal stromal tumors (GIST) can develop anywhere in the gastrointestinal (GI) tract.  Neurofibromatosis 1 (NF1) is a genetic disorder that causes tumors to form on nerve tissue and is usually diagnosed in children and young adults. Patients with NF1 have an increased risk of developing GIST. Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch, is leading a study to see how NF1-related GIST responds to treatment with selumetinib, a protein kinase inhibitor that blocks a protein called MEK. Dr. Widemann’s team recently demonstrated that selumetinib shrinks other NF1 tumors called plexiform neurofibromas by blocking the MEK protein. Many types of cancer cells express high levels of protein kinases, which play an essential role in the growth and spread of cancer cells. Investigators hope that by targeting MEK there will be an impact on the growth of NF1-related GIST and tumor size. For more information on this trial, visit:

Posted on Mon, 05/15/2017