Researchers from Mass General Cancer Center’s Central Nervous System Metastasis Center have devised a way to shrink brain tumors by up to 90% using already existing drugs. The researchers used two cancer drugs approved to treat melanoma to treat papillary craniopharyngioma (PCP), a rare type of brain cancer with a 10-year average survival rate.
The researchers recruited 16 patients who had not gone through radiation therapy and gave them cancer drugs targeting the proteins MEK and BRAF, which play a role in cell growth. Surprisingly, the drugs were more than 90% effective at shrinking tumors even though they are only approved to treat melanoma.
Central Nervous System Metastasis Center director and lead study author Priscilla Brastianos said that the response was “unprecedented” and represented the highest response rate so far in brain tumor systemic therapy.
Brain tumors tend to have lower survival outcomes because the blood-brain barrier often prevents various types of chemotherapy from reaching tumor cells in the brain. Treatment via surgery can also be difficult depending on the location of the tumor.
Published in the “New England Journal of Medicine,” the recent study is the first to demonstrate the effectiveness of medical therapies in brain-tumor treatment. The findings could help to significantly improve treatment outcomes for PCP patients and even encourage more research into the development of advanced precision medicine to treat brain tumors.
Current treatment protocols for PCP typically involve surgery and radiation, which can be challenging as PCPs are often located next to crucial vascular and brain structures. As a result, PCP patients can suffer from “lifelong complications due to surgery and radiation,” said Brastianos.
According to Pennsylvania State University professor of neurosurgery and oncology Michael Glantz, surgery and radiation treatment for papillary craniopharyngioma can dysregulate hunger and impair intellectual function, vision and sleep, among other side effects.
The challenges involved in treating PCPs coupled with the potential for lifelong side effects are what inspired Brastianos and his team to find safer and more effective treatments for PCP.
McGill University Department of Biochemistry professor and Goodman Cancer Institute member Ian Watson said the study’s results were “incredibly impressive.” Although he wasn’t involved in the study, Watson said that the therapy was even more promising because none of the participants developed progressive disease during treatment.
However, Weill Cornell Medicine professor of neurosurgery Ted Schwartz notes that the study had several limitations. For instance, 14 of the participants experienced serious side effects during the study, and PCP continued to progress in three patients after the study ended.
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