Drug trial perplexities

The drug trial described in this New York Times article is one for which I may qualify, should I need it eventually. We talked about this so-called B-RAF inhibitor at my consultation last week with Dr. C, who mentioned that Providence will soon be joining the trial. To qualify, I would need to have evidence of tumors, which I won’t have after surgery on Thursday, plus the specific genetic mutation that this drug is designed to negate.

Dr. C called the drug (PLX4032) “an agent of high promise,” but he’s been around long enough to have said that before about other experimental drugs. He’s wary for good reason, as there have been no new drugs approved for advanced melanoma in the last 12 years. As alluded to in this article, there is also the matter of how long-lasting the responses to the drug will be. I hope that the patients who are enrolled have been told that their remissions are not likely to be long-term. Some have been so sick they’re undoubtedly grateful just for a reprieve.

One more thing: All patients in a clinical trial like this are “randomized” for the drug being investigated, meaning there’s a 50/50 chance a patient won’t get it. The PLX4032 trial is randomized with dacarbazine, a chemo drug with nightmarish side-effects. The best scenario for me would be to never need any of these drugs, but if I do, that the phase 3 trial proves successful enough that the FDA quickly grants approval. The good news is that the current gold standard for systemic therapy—interleukin-2—sets a pretty low standard for new drugs currently in the pipeline.

The NYT article, by the way, is the second in a three-part series. Here's the link to part one. It's a lot of detail about a single drug, but also includes useful background on melanoma in general.