Treatment options for melanoma have advanced in the past year with heartening results. As we wrote in March 2011, the drug Yervoy (ipilimumab or “ipi”) appears to be successful for a small group of patients with inoperable, metastatic melanoma in extending survival. In fact, WebMD says that “Yervoy … is the first drug ever shown to help late-stage melanoma patients live longer.”
Now, there’s more news to be hopeful about on the pharma front with potentially greater results: The FDA has also given the go-ahead to a drug called ZELBORAF (vemurafenib, pronounced vem-yoo-RAF-en-ib). A comprehensive overview by ABC News offers a clear explanation of how Zelboraf attacks a genetic mutation (known as BRAF V600E) which is found in about half of melanoma patients, inhibiting the disease’s ability to spread.
We now have the capability to analyze a patient’s melanoma tumor for the genetic mutation BRAF and use the targeted treatment Zelboraf to attack the tumor, shrink it and stop the progression of this deadly disease,” said Dr. Anna Pavlick, director of the NYU Melanoma Program at the NYU Cancer Institute, who has been involved in clinical trials for Zelboraf. …Zelboraf shuts down the abnormal signals of the tumor cells that are caused by the genetic mutation and stops the cells from dividing, without affecting healthy cells.
At the same time, the FDA approved a genetic test to determine if patients carry the mutation since only those with the abnormal “BRAF” gene can take Zelboraf. Interestingly, since the same genetic mutation is found in those with other forms of cancer, there may be future help from this drug beyond skin cancer and is now, for example, being tested on thyroid cancer patients.
This is the fastest the FDA has ever approved a drug to come to market – in just five years. And even better: Zelboraf, which is a first-in-class drug, is anticipated to be available in the next two weeks.
There are differences between Yervoy and Zelboraf. As described in the coverage by the SF Chronicle,
Yervoy was found to extend patients’ lives, an improvement over many current treatments. However, the drug works for less than 20 percent of patients, and doctors say they can’t predict which patients will find it most effective.
Zelboraf was clinically effective in 50 percent of patients [with the] specific genetic mutation … Most of the therapies for melanoma work for less than 20 percent of patients, and some fall into single digits.
Neither Zelboraf nor Yervoy cure melanoma. And as a patient who had great success in a clinical trial for the new drug said, “there’s nothing that says this medication will help you forever.” But these drugs bring hope for a longer life, and as new therapies come along the melanoma pipeline, there is reason to be optimistic.





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