The activating BRAF V600E mutation has been successfully targeted with dual molecular therapy in melanoma, non–small cell lung carcinoma (NSCLC), and thyroid cancer. BRAF V600E mutations have also been identified in a subset of patients with pediatric and adult brain tumors, and several case reports have indicated the potential efficacy of targeted therapy in these patients. To date, these studies have mostly been performed using single-agent BRAF inhibitor therapy in recurrent low-grade gliomas (LGGs; Table 1). This report describes the use of combination dabrafenib (BRAF inhibitor) and trametinib (MEK inhibitor) in 2 adults with high-grade gliomas (HGGs). The first patient was treated at time of diagnosis, whereas the second was treated at time of recurrence in conjunction with the antiangiogenic agent bevacizumab. Both patients showed rapid and dramatic clinical and radiographic responses.
Dr. Ansstas has disclosed that he is a member of the advisory board and speaker's bureau for Novocure. The remaining authors have disclosed that they have no financial interests, arrangements, affiliations, or commercial interests with the manufacturers of any products discussed in this article or their competitors.
Drs. Johanns and Grierson are supported by the NIH T32 HL007088 training grant through the Division of Hematology and Physician-Scientist Training Program at Washington University School of Medicine.
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