A targeted treatment has had unprecedented success in shrinking a rare brain tumor, according to clinical trial results. These tumors are papillary craniopharyngiomas (PCPs), and the drug combo is called vemurafenib/cobimetinib. Each of 15 patients who received one or more cycles of targeted therapy responded to treatment, with an average 91% reduction in tumor size, Mass General Cancer Center researchers reported. PCPs can cause substantial health problems. They are typically treated with surgery and radiation, but incomplete removal of the tumor and toxicity from radiation can leave patients with lifelong health challenges after treatment. This can include neuroendocrine dysfunction, as well as vision or memory loss. “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,” said study co-author Dr. Priscilla Brastianos, director of the cancer center’s Central Nervous System Metastasis Center. “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,” Brastianos added in a hospital news release. This was the first multicenter treatment protocol in this rare tumor, according to Mass General Cancer Center, which led the study. The findings were based on laboratory discoveries by researchers who studied the genetic drivers… read on > read on >







