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New drug shows great promise for deadly melanoma

Trametinib, a new drug for treating melanoma, is showing promise in clinical trials. NBC's Robert Bazell reports.

An experimental new drug called trametinib is showing great promise in treating the deadliest of skin cancers, advanced melanoma.

In data presented at the annual meeting of The American Society for Clinical Oncology, from a trial that followed more than 300 patients, those who took trametinib appeared to do better than those who used traditional chemotherapy.

Median time of survival with no progression of cancer was 4.8 months in the trametinib patients, compared with 1.5 months for those on the traditional regimen. That might not sound like much, but such results often indicate that patients with less-advanced cancer will do far better on the drug than the volunteers in the trial. The results are good enough that the drug’s manufacturer, GlaxoSmithKline, will apply to the federal Food and Drug Administration for approval.

That news followed a report two days earlier in which scientists showed that a more experimental drug called PD-1 antibody could boost the immune system and help patients with advanced melanoma, as well as those with lung and kidney cancer. That drug, however, is still years away from heading for the FDA.

These individual results are part of a much bigger picture. In the vast majority of cases, melanoma is caught early and cured by surgery. But if it returns it can be deadly -- and until recently it almost always was. But in the past year and a half, the FDA has approved new drugs for advanced melanoma. There’s Zelboraf,  which, like trametinib, targets an aberrant gene that drives the cancer. And  there’s Yervoy, which boosts the immune response. Several other experimental drugs using both approaches are in the pipeline.

Experts like Dr. Keith Flaherty of Massachusetts General Hospital say the care of patients with advanced melanoma has changed drastically. Eighteen months ago, he told me that the best hope for patients was to participate in a clinical trial with experimental therapy.

“Now,” he added, “we have multiple new treatments which we know can be game-changers in a profound way – and in the long-term. So it really does give us a sense of control -- doctor and patient -- over a circumstance over which we never had control.”

One of the things doctors are trying to figure out is in what combinations or in what sequences they can best use the new drugs. But there is a big problem. Companies price these drugs at tens of thousands of dollars, sometimes more than $100,000 a year. Many doctors and policy experts are asking whether the medical system can continue to withstand these charges.

At the same time, cancer doctors are still experiencing frightening shortages of older, very inexpensive drugs which keep many patients alive. The FDA says it has no power to tell companies which drugs to make. But it’s clear the shortages of the cheap drugs and the enormous costs of the new experimental ones are both sources of huge concern

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