Anti-CTLA-4 and BRAF inhibition in patients with metastatic melanoma and brain metastases

Until recently, there were few treatment options for patients with advanced or metastatic melanoma associated with significant increases in overall survival. Of these, patients with disease that has metastasized to the brain have a particularly poor prognosis and generally ineffective treatment options. Recent advances in immuno-oncology have led to the approval of ipilimumab, an antibody that blocks cytotoxic T-lymphocyte antigen-4 to augment antitumour T-cell responses, and vemurafenib, a BRAF kinase inhibitor. These agents, along with others on the horizon, are promising treatment options for patients with melanoma and CNS involvement. Here we review the data generated to date in patients with melanoma and brain metastases and suggest the direction that future studies may take to optimize outcomes in this subpopulation of patients.