Scientists at Harvard Medical School have developed a new treatment for papillary craniopharyngiomas, a rare brain tumor. The therapy has shown unprecedented success in shrinking tumors. Research published New England Journal of Medicine.
The study included 16 patients, 15 of whom completed at least one 28-day treatment cycle with the combination of vemurafenib and cobimetinib. Over four cycles, the mean reduction in tumor size ranged from 68% to 99% (mean 91%).
After discontinuation of experimental treatment, seven patients received no further treatment. Six showed no evidence of tumor progression when followed for about two years. During the treatment, none of the patients had any tumor growth or died.
“All patients who completed one or more cycles of therapy responded to therapy with the highest response rate of any medical therapy for brain tumors,” the scientists said.
The authors’ previous work has shown that approximately 95% of papillary craniopharyngiomas have mutations in the BRAF gene, which is also found in some forms of melanoma. Scientists have found that existing cancer drugs, BRAF/MEK inhibitors, can eradicate gene-defective papillary craniopharyngiomas.
The researchers noted that more research is needed to determine the optimal number of treatment cycles.
Papillary craniopharyngiomas are usually treated with surgery and radiation, but incomplete tumor resection and radiation toxicity can lead to vision or memory loss.
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