Pair of Melanoma Studies Usher in a New Standard of Therapy for Patients with Advanced Disease

Patients with advanced melanoma are finally seeing the benefits of research. At the plenary session, investigators revealed positive results of two advanced melanoma studies. The first presentation showed patients who received the immunotherapy drug Yervoy (ipilimumab) plus dacarbazine survived longer than patients only on dacarbazine, a decades-old chemotherapy that has limited success against the disease. While Yervoy was approved in March for patients who have progressed on prior treatments, this most recent phase 3 study shows the drug is useful for previously untreated patients, improving median overall survival from 9.1 months to 11.2. Median duration of response more than doubled from 8.1 months with dacarbazine to 19.3 months with the Yervoy combination. Side effects were similar to prior studies, which include rash and diarrhea.

The second study compared dacarbazine with the oral investigational drug vemurafenib (PLX-4032), which inhibits a specific BRAF protein mutation found in about half of all melanoma tumors. The BRIM3 study looked at 675 patients with BRAF-positive tumors and found that vemurafenib extended survival by 63 percent, improving the median six-month survival rate from 64 percent on dacarbazine to 84 percent. The median progression-free survival was also better with vemurafenib. It took 1.6 months for half of the dacarbazine group to have cancer progression, whereas 5.3 months passed before half of the vemurafenib group had seen their cancer grow—an improvement of 74 percent. Side effects of vemurafenib included rash and low-grade cutaneous squamous cell carcinomas, which are benign cancers removed by a dermatologist. Due to the promising results, the drug is expected to be submitted for Food and Drug Administration approval by the end of the year.

The studies were presented on the heels of an announcement that the two drugs will be examined as a combination therapy in an early-phase trial.

Correction: An earlier version of this article incorrectly stated the submission for vemurafenib was expected later this year.Vemurafenib was submitted for approval in May.