MGH/Harvard-Led Team Reports First-Ever Successful Systemic Treatment of Recurrent, BRAF-Mutant Brain Tumor (Craniopharyngioma) with Monoclonal Antibody BRAF Inhibitor Dabrafinib

A team led by Massachusetts General Hospital (MGH) investigators has reported the first successful use of a targeted therapy drug to treat a patient with a debilitating, recurrent brain tumor. In a paper published online on October 23, 2015 in the Journal of the National Cancer Institute (JNCI), the researchers report that treatment with the BRAF inhibitor dabrafinib led to shrinkage of a BRAF-mutant craniopharyngioma that had recurred even after four surgical procedures. More than a year after dabrafinib treatment, which was followed by surgery and radiation therapy, the patient's tumor has not recurred. The article is titled “Dramatic Response of BRAF V600E Mutant Papillary Craniopharyngioma to Targeted Therapy.” "This is the first time that a systemic therapy has shown efficacy against this type of tumor," says Priscilla Brastianos (photo), M.D., co-lead author of the JNCI report. Dr. Brastianos is Director, Central Nervous System Metastasis Program. Massachusetts General Hospital. Harvard Medical School. She is also Instructor of Medicine, Harvard Medical School. "This has the potential of completely changing the management of papillary craniopharyngiomas, which can cause lifelong problems for patients - including visual defects, impaired intellectual function, and pituitary and other hormonal dysfunction." Craniopharyngiomas are pituitary tumors that, while technically benign, can cause serious problems because of their location near critical structures, such as optic and other cranial nerves and the hypothalamus. Not only does the growing tumor compromise neurological and hormonal functions by impinging on these structures, but treatment by surgical removal or radiation therapy can produce the same symptoms by damaging adjacent tissues.
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