Exceptional Response in Brain Tumor Patient Instills Confidence in Personalized Medicine

Source: AJMC, November 2015

A young man, less than 40 years old, with recurrent craniopharyngioma (pituitary tumor) harboring a BRAF V600E mutation, showed a dramatic response when treated with BRAF V600E inhibitors—85% reduction in tumor volume. This case has the “potential of completely changing the management of papillary craniopharyngiomas,” according to study author Priscilla Brastianos, MD, of the Massachusetts General Hospital (MGH) Cancer Center.

The study, published in the Journal of the National Cancer Institute (JNCI), reports on the case study of a patient who came to the emergency department at MGH, 7 months after having undergone surgery to excise a brain tumor. A CT scan of the patient, who complained of confusion, impaired vision, severe headaches, and vomiting, revealed a 4 cm cystic tumor. Six weeks following partial removal of the tumor, the patient was brought back and underwent additional surgery. At this point, the tumor was confirmed to carry a mutant BRAF. Another unsuccessful surgery and a recurrence in 2 weeks prompted the providers handling the case to treat the patient with the BRAF-V600E inhibitor dabrafenib, currently approved for the treatment of patients with unresectable or metastatic melanoma harboring the BRAF-V600E mutation…..