The experimental oral drug selumetinib is the first systemic drug to show significant clinical benefit in patients with metastatic uveal melanoma, according to clinical trial results presented at the 2013 Annual Meeting of the American Society of Clinical Oncology in Chicago.

A novel drug called selumetinib, if approved, would be the first oral drug to effectively treat uveal melanoma (above). Photo: Paul M. Karpecki, OD
In this Phase II study of 98 patients with metastatic uveal melanoma, researchers randomly assigned 47 patients to receive selumetinib and 49 patients to receive temozolomide, the current standard chemotherapy agent for skin melanoma. In the selumetinib group, 50% of patients experienced statistically significant tumor shrinkage, with 15% achieving major shrinkage. By comparison, no patients in the temozolomide group achieved significant tumor shrinkage.

In addition, selumetinib was shown to control tumor growth more than twice as long as temozolomide—for nearly 16 weeks vs. seven weeks.

Although uveal melanoma is rare—only 2,500 cases are diagnosed in the US each year—about half of patients diagnosed eventually develop metastatic disease. There is currently no drug approved specifically for treatment of this cancer.

“This is the first study to show that a systemic therapy provides significant clinical benefit in a randomized fashion to advanced uveal melanoma patients, who have very limited treatment options,” says lead investigator Richard D. Carvajal, MD, a medical oncologist at Memorial Sloan-Kettering Cancer Center in New York. “This clinical benefit has never been demonstrated with other conventional or investigational agents, which is all we have been able to offer patients for decades.”

Dr. Carvajal is now planning a multicenter, randomized trial to attempt to repeat the drug’s effect. “If we can confirm selumetinib’s effectiveness in treating advanced uveal melanoma in this follow-up trial, it will become the standard therapy for this disease.”