Vemurafenib Promising Against Deadly Melanoma

VIDEO: Dr. Kevin Kim says vemurafenib showed a high percentage of tumor shrinkage.
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When Richard Kaminski had an unusual mole removed from his cheek in 1998, he thought it was the end of his experience with melanoma.

But more than 10 years later, Kaminski developed a cough that didn't go away. Medical tests confirmed the diagnosis: metastatic melanoma that had spread to his lungs.

Melanoma is a form of skin cancer that kills 85 percent of its victims within five years if it has spread. It is responsible for about 9,000 deaths in the United States a year, according to the American Cancer Society.

Kaminski was floored. "I had this awful thing working in my lungs," he said.

Doctors treated Kaminski with a drug typically used against this form of cancer, but without success. It was only when his oncologist put him in touch with Dr. Anna Pavlick at New York University, who enrolled him in a clinical trial of a medication called vemurafenib, that he began to turn the corner on the deadly illness.

Before treatment, Kaminski recalled, "I had great difficulty breathing. I couldn't put sentences together because I couldn't get a deep breath. I had pain in my chest." Three weeks after beginning the drug, "A lot of that was greatly diminished," he said.

Within three months, Kaminski's symptoms disappeared. Scans showed his tumors starting to regress. By the end of 2010, the tumors were gone.

Kaminski, now 65, is understandably thankful.

"In two weeks, I will be two years on this drug," he said. "It was a lifeline."

On Wednesday, the results of the clinical trial in which Kaminski was enrolled appeared in the New England Journal of Medicine. In the study conducted at 13 centers in the U.S. and Australia, researchers followed 132 patients with Kaminski's type of melanoma who had failed to respond to standard treatment. In about half of them, vemurafenib shrunk their tumors. For another third of the patients, the tumors showed no progression once the drug regimen had kicked in. Dr. Lynn Schuchter at the University of Pennsylvania, another of the study's authors, called these numbers "unprecedented."

"I've treated patients [with melanoma] for almost 25 years and never seen a drug with this kind of activity," Schuchter said. "It's so much better than the therapies that have been available to us before."

Also impressive was the improvement in survival; patients on the drug lived, on average, for an additional 15.9 months after treatment began, compared with the six to 10 months typically seen with the disease. A larger trial, also published this year in the New England Journal of Medicine, showed that the drug brought about improved survival at six months. But the authors of the new study were able to follow some of their patients much longer -- for more than a year after they'd started treatment.

"[They] really show much prolonged survival," Schuchter said, "and that's been my experience clinically as well, that they're living longer and with high quality of life."

For patients like Kaminski who fail standard treatment, the new drug offers hope. Unlike many other cancer drugs, vemurafenib was just as effective in patients who had failed a previous treatment as it was in patients who had received the drug right off the bat -- a rare finding when it comes to cancer treatments.

The drug is not without its limits. In targeted therapy, cancer cells can mutate slightly and stop being a target, a process called resistance. In this study, cancer tended to recur in patients after about seven months of treatment. Dr. Kelly McMasters, chairwoman of surgery at the University of Louisville who treats melanoma patients, points out, "It can cause the tumors to shrink, but they will recur on average in about six months."

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