As reported in Neuro-Oncology Magazine (January 12th, 2017), with leadership from the Dana Farber Cancer Institute’s Dr. Charles Stiles and up and coming researcher, Dr. Sarah Buhrlage, ground-breaking science has identified a type II RAF inhibitor that has good brain penetrance and attacks the very antagonist (BRAF V600E, KIAA1549) that is responsible for tumor growth. It has been known that while mutations in the BRAF gene (which represents roughly 75% of pediatric low-grade astrocytomas) are responsible for tumor growth, the drugs that are meant to target the mutation are not sufficient enough to cross the blood-brain barrier and therefore are not able to stop the tumor from growing. But, with this study, a panel of small molecule RAF inhibitors was screened for drugs that would cross into the brain and target PLGA cells expressing KIAA1549. The outcome of this discovery will enable clinicians and researchers to launch the first type II clinical trial for PLGA patients using compound MLN2480 to target the tumor and arrest growth. Funding for this study was provided by A Kids’ Brain Tumor Cure Foundation and a number of other like-minded non-profit organizations. The clinical trial that will test the efficacy of MLN2480 in pediatric patients is being conducted by the Pacific Pediatric Neuro Oncology Consortium (PNOC) in partnership with Dr. Mark Kieran from the Dana Farber Cancer Institute. This trial is projected to open for enrollment in the spring of 2017. Funding for this trial will be provided by AKBTC in partnership with Team Jack and a number other non-profit organizations.
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