Melanoma/Skin Cancer Health Center

New Drug May Treat Advanced Melanoma

New Melanoma Drug: Making It Better

Yes, the drug -- a pill taken twice daily -- has side effects. The most common ones are a rash, sun sensitivity, joint discomfort, and fatigue. A number of patients also developed non-deadly squamous-cell carcinomas on the skin. Flaherty says these side effects tend to be moderate and manageable -- and that patients have a good quality of life while on therapy.

The drug's most important drawback is that it isn't a cure. Eventually, tumor cells find new ways to grow and become resistant to the new drug.

"Nine months extra progression-free survival is not what patients come to us for," Flaherty says. "But we think there are ways to make it better."

"While we need a lot of research, there are many achievable ways to combat that resistance," Sondak says. "Once we understand it a little better and develop new drug combinations, we can keep moving the bar and get responses in more people that last longer and longer. Maybe, eventually, even to the point where we cured this person."

New Melanoma Drug: Turning Point for Cancer Research?

Sondak says that the new drug's success has huge implications for the entire field of cancer research.

"What I think is exciting is the broad perspective: Melanoma isn't the only cancer with this mutation, just the one where it is most common," he says. "And what's really good news is the success of this idea that you can target a cancer gene and do something good. Now we can start looking for similar mutations in other genes important in other cancers. We now have another proof that this is a fertile line of research."

Today's exciting results come just eight years after the first genetic study found that the BRAF mutation is important for tumor growth.

"So in a very short time, from discovery of the mutation to publication of the Flaherty trial results, we went through all those individual steps and went very, very quickly to a point where everyone could see we had a drug with much better responses than any other melanoma treatment," Sondak says. "To me that is the excitement. It shows the cancer research we are doing is paying off."

What Should Today's Melanoma Patients Do?

Despite all this excitement, the reality is that PLX4032 is not yet ready for prime time. A phase III clinical trial is now enrolling patients with BRAF-positive tumors. In that trial, some patients will get the drug (in a version dubbed RO5185426) and others will get standard treatment.

Only after this trial is completed and analyzed will it be known whether the drug truly works -- and only then will it be available to patients.

There's a bit more good news for the future. A similar drug from GlaxoSmithKline, called GSK2118436, is enrolling patients in a phase II study.