Unprecedented success in treating a rare brain tumor with drugs that inhibit the BRAF/MEK gene

by Anika Shah - Technology
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A drug treatment based on precision medicine has shown some unprecedented results in the therapy of a rare type of brain tumor, papillary craniopharyngiomas (PCP), which may represent a paradigm shift in the approach to this type of tumors, which account for between 1.2-4.6% of intracranial tumors

The study, which has just been published in The New England Journal of Medicine, has been carried out by researchers at the Mass General Cancer Center, belonging to the Mass General Brigham health care system. It’s about a phase II trialin which they have been included 15 patients. One of the most striking results is that 94% of patients responded to therapy.

Papillary craniopharyngiomas are a rare type of brain tumor that cause significant morbidity in patients. Although surgery and radiation are often used to treat these tumors, incomplete removal of the tumor and radiation toxicity can leave patients with lifelong health problems after treatmentincluding neuroendocrine dysfunction or loss of vision or memory, the study authors explain.

“The tumor is located in a delicate zoneclose to the hypothalamus and the pituitary, very enervated and acting on it to try to eliminate the tumor with radiotherapy and surgery carries significant risks”, he explains in his assessment of the study, Paul SantamarinaPhD in Biomedicine from the University of Oviedo and member of the Cancer Epigenetics group, attached to the Institute of Health Research of the Principality of Asturias, the CSIC and the IUOPA.

Researchers at the Mass General Cancer Center have conducted the first multicenter treatment protocol for this tumor considered rare due to its frequency.

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