
Exceptional Response in Brain Tumor Patient Instills Confidence in Personalized Medicine
Treatment of a recurrent brain tumor harboring a BRAF mutation with an inhibitor approved for melanoma resulted in dramatic tumor shrinkage.
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,”
The study, published in the
A dramatic response was seen within 4 days of treatment, with a 25% tumor shrinkage, the authors report. Following a 50% shrinkage in tumor by day 17, the authors added a MEK inhibitor, tramatenib, also
“It is quite remarkable how quickly we have been able to go from identifying the genetic driver of papillary craniopharyngiomas to testing the idea in a patient that needed help. It was only last year that, along with Dr Brastianos and colleagues, we first described in Nature Genetics that nearly all papillary cranionpharyngiomas have mutations in BRAF,” said co-senior author Sandro Santagata, MD, PhD, of Brigham and Women's Hospital's Pathology Department.
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