Researchers at Boston-based Mass General Cancer Center found a treatment that reduced a rare type of brain tumor by an average of 91 percent in a clinical trial.
The phase 2 trial, published in the New England Journal of Medicine, treated 16 patients with papillary craniopharyngiomas with a BRAF/MEK inhibitor using a vemurafenib/cobimetinib drug combination. Over the course of four cycles, tumors were reduced between 68 percent and 99 percent. Seven patients received no other treatment after continuing the drug and six had no evidence of tumor progression nearly two years later.
However, three patients discontinued treatment after experiencing adverse events, including one who dropped out after eight days due to anaphylaxis and acute kidney injury. The treatment also caused rashes in six patients.
"All patients who completed one or more cycles of therapy responded to treatment, which is the highest response rate to date of any medical therapy for brain tumors," study author Priscilla Brastianos, MD, director of the Central Nervous System Metastasis Center within the cancer center, said in a news release. "These unprecedented results signal a paradigm shift for targeting brain tumors because they show that, with the right target and the right drugs, precision medicine can have a dramatic impact on brain tumors."
Papillary craniopharyngiomas are a rare type of brain tumor that cause substantial morbidity for patients. Treatment often involves surgery and radiation, but many patients end up with life-long health challenges following treatment including vision and memory loss.