Clinical trial studies drug for pediatric diffuse intrinsic pontine glioma (DIPG) treatment

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Diffuse intrinsic pontine gliomas (DIPGs) are the leading cause of brain tumor deaths in children. They develop in the lowest, stem-like part of the brain, called the pons, which controls vital functions such as breathing, blood pressure and heart rate. DIPG is a high-grade tumor that is fast-growing and spreads throughout the brain stem. It is hard to treat and has a poor prognosis. Panobinostat, a histone deacetylase inhibitor, blocks some of the enzymes that tumor cells need to grow and spread. Katherine Warren, M.D., of the Pediatric Oncology Branch is leading a pediatric clinical trial as part of a multicenter study sponsored by the Pediatric Brain Tumor Consortium. The goal of this trial is to determine the safety and best dose of panobinostat for patients with DIPGs. According to Dr. Warren, “This is the first trial to translate extensive DIPG-specific pre-clinical information to the clinic, and hopefully, it will translate into improved outcomes for patients with this devastating disease.”

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Summary Posted: 01/2018