Withholding lifesaving drugs to prove a point

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The front page of Sunday’s New York Times featured a heartbreaking story about two cousins, both suffering from widespread melanoma, a lethal type of skin cancer. Both young men’s melanoma had a specific gene mutation called B-RAF, which is specifically targeted by a new drug being tested by Roche Pharmaceuticals. Called PLX4032, the drug seems to be highly effective, stopping tumor growth in 81 percent of patients with the mutation for an average of eight months. The standard chemotherapy treatment, a 40-year-old drug called dacarbazine, slows tumor growth in only 15 percent of patients for only two months, and is highly toxic as well.

But because Roche needs to complete a Phase III trial to prove the drug’s efficacy, only one of the cousins was given the drug; he showed dramatic improvement. The other was randomly placed in the control group, received standard chemotherapy — and eventually died.

Dr. David E. Fisher, a leading melanoma biologist at Massachusetts General Hospital, tells the Times that the controlled trial is “nuts. I don’t know anyone who hasn’t shuddered at the concept that we can’t let patients on the control arm cross over because we need them to die earlier to prove this point.”

Dr. Bloom agrees. “Clinical trials are stopped all the time for positive and negative reasons. I don’t see why this should be any different. It makes no sense. If there was ever a time to stop the trial and give everyone with the mutation the drug, this would be it. Not to do so is almost like condemning such patients to death, given the relentless predictability of metastatic melanoma.”