2011 Annual Report: First Effective Treatments for Advanced Melanoma

In March 2011, the US Food and Drug Administration announced its approval of the drug ipilimumab (Yervoy™) for the treatment of patients with metastatic melanoma. It is the first drug ever shown to improve overall survival for patients with advanced melanoma.

Ipilimumab uses an approach known as immunotherapy, which exploits the body’s own immune system to attack cancer. The early development of this specific type of immunotherapy, originally known as anti-CTLA-4, was led by a Memorial Sloan Kettering immunologist, and Memorial Sloan Kettering researchers led preclinical and clinical studies using ipilimumab.

In August, another drug, called vemurafenib (Zelboraf®) — a therapy that targets a mutation in a gene called BRAF, which is present in about half of all melanomas — was also approved by the FDA for the treatment of advanced melanoma. Again, Memorial Sloan Kettering physician-scientists led clinical and preclinical studies of the therapy and headed a large phase III clinical trial that resulted in the drug’s approval.

Then, in a study published in December, Memorial Sloan Kettering researchers reported that they had uncovered a previously unknown mechanism by which tumors can become resistant to vemurafenib. The hope is that their findings will lead to the development of new treatments and will inform the design of next-generation drugs that are not as susceptible to this form of resistance.