Melanoma brain metastases and vemurafenib: Need for further investigation

Nicole M. Rochet, Roxana S. Dronca, Lisa A. Kottschade, Rahul N. Chavan, Brian Gorman, Julie R. Gilbertson, Svetomir N. Markovic

Research output: Contribution to journalArticlepeer-review

36 Scopus citations


Brain metastases are a major cause of morbidity and mortality in patients with advanced melanoma. With the development of targeted agents for the treatment of metastatic melanoma, a great deal of interest has focused on whether selective BRAF inhibitors may play a role in the treatment of brain metastases in lieu of or in addition to surgery and/or radiation therapy. However, relatively little is known about the intracranial effectiveness of vemurafenib, the only US Food and Drug Administration-approved selective BRAF V600E inhibitor, because patients with brain metastases have historically been excluded from vemurafenib clinical trials. We describe 3 patients with BRAF V600E mutation metastatic melanoma in whom treatment with vemurafenib resulted in prompt extracranial disease response but progression of metastatic disease in the brain. Further, we discuss possible mechanisms responsible for the suboptimal central nervous system response observed in these patients and alternative therapies for patients with melanoma metastatic to the brain.

Original languageEnglish (US)
Pages (from-to)976-981
Number of pages6
JournalMayo Clinic proceedings
Issue number10
StatePublished - Oct 2012

ASJC Scopus subject areas

  • General Medicine


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