Yesterday's New York Times front page had a story by Amy Harmon that made me cry. It was about two cousins in their early 20s with deadly melanoma who were enrolled in a controlled clinical trial of a new drug, PLX4032, that dissolved tumors in patients with a certain gene mutation. But because of the rules of clinical trials, only one cousin, Thomas McLaughlin, got the new drug that shrank his tumors and has given him some extra time to be a dad to his two kids. His cousin, Brandon Ryan, was given the old chemo that doesn't stop the spread of tumors. He died at the age of 22. From 30 years of experience, doctors know that the old drug is basically useless. But being in a controlled trial means that the participants are randomly assigned either the old drug or the new one in order to find out its benefits or risks. So the young man on the old drug died within a month of being denied the new drug by a computer selection.
Rules are rules, say some doctors, but others say in the fast-changing world of research and medication, the FDA needs to revise the protocol. That seems to be a no brainer, and if the rules could even have been bent a little, perhaps Brandon Ryan wouldn't have had to die a painful death at age 22 in the name of science. His cousin has so far survived nine months on the new drug and is working again..
As the widow of a cancer patient, I felt despair and moral outrage at the heartlessness of this. I remember a stupid problem I had in the hospital when Peter's doctors wanted him to take a drug that was not yet approved for his myeloma. I had hired a private duty nurse who refused to give it to him because the label warned that it could cause infertility. The nurse was probably 65 if she was a day, and it was frustrating, but it was a minor irritant on the scale of what the mother of Brandon Ryan went through. As she was quoted in the Times: "What gives them the right to play God? It doesn't make sense to say, 'We want you for a statistic' instead of giving them a chance at life."
Clearly it's time the rules changed as newer and targeted drugs are being developed. If two new cars were developed and one buffered the driver in a crash 80% more than the other, would the public be expected to try their chances in the unsafe car just to prove the effectiveness of the safe one? It's time the FDA changed its rules so that the Brandon Ryans of this world don't die without having a fighting chance. I'm no scientist but from what I've read, PLX4032 sounds as if it works to prolong life with less pain even if it is only for a few months.
Think of how many beautiful sunsets and walks in the park you can enjoy in a few months. Maybe it gives the patient a chance to see his child take a first step or to graduate college. How do human beings in the name of science deny this?