
A multi-institutional study has identified a new treatment option for papillary craniopharyngioma, a rare form of cancer that forms along the central nervous system.
“Brain tumors are rare, and papillary craniopharyngioma tumors are rare among this group,” said Adam Cohen, M.D., co-director of the neuro-oncology program at the Inova Schar Cancer Institute. “We found that a drug therapy approved for another cancer worked beyond all of our expectations, and some people whose tumor shrank after treatment continue to do well without radiation or surgery.”
Papillary craniopharyngiomas grow deep within the brain and form tumors along critical structures, like the nerves connected to the eyes, memory circuits of the brain, and the hypothalamus that controls the production of hormones that control growth, development, and metabolism. These tumors affect primarily the very young and very old.
Immunotherapy and chemotherapy are ineffective for papillary craniopharyngiomas. Surgery and radiation are both effective but can produce both short- and long-term complications, like endocrine malfunction, vision problems, and impaired intellectual function. In addition, most patients experience recurrence of disease after treatment.
Papillary craniopharyngiomas are genetically simple tumors and are dependent on single-driver mutations that are less prone to develop resistance to drug therapies. Most craniopharyngiomas have a mutation on the BRAF V600E gene.
Vemurafenib and cobimetinib are two drugs approved by the FDA that target the BRAF V600E mutation for a variety of cancers, like melanoma, lung, and thyroid cancer.
“This mutation is fundamental for the formation and growth of these tumors,” said Cohen. “It is difficult to get drugs into the brain, and we wanted to prove the concept that these targeted drugs will work for tumors in the brain.”
Cohen participated in a study led by researchers at the Massachusetts General Hospital Cancer Center and the Mayo Clinic. The results of their study was published in the July issue of New England Journal of Medicine in an article titled “BRAF–MEK Inhibition in Newly Diagnosed Papillary Craniopharyngiomas.”
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