Celebrating a promising Phase II trial for a rare brain tumor treatment

A New England Journal of Medicine article, “BRAF–MEK Inhibition in Newly Diagnosed Papillary Craniopharyngiomas”, describes a Phase II clinical trial and offers some insight into potential targeted treatments for a rare cancer. Papillary Craniopharyngiomas typically occur in the region of the brain near the pituitary gland and optic nerves. Radiation and surgical treatment are often associated with substantial morbidity such as vision loss, neuroendocrine dysfunction, and memory loss. The study team explored whether treatment BRAF–MEK inhibitor combination vemurafenib would lead to better patient outcomes.

For Dr. Sandro Santagata, this work closes a chapter – in 2013, Santagata co-led a team that identified two unique subtypes of craniopharyngiomas and found that papillary craniopharyngiomas nearly always have BRAF-V600E mutations. This discovery stimulated other efforts to repurpose drugs developed for melanoma, resulting in this Phase II clinical trial, in which 15 of 16 patients had a partial response or better to the BRAF-MEK inhibitor. On LinkedIn, Sandro reflected on how this paper represents not only an important step in treating rare cancers but exemplifies how contributions from clinicians, patients, pathologists, philanthropists, and clinical trial experts can move medicine forward.

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