By all rights, Gerald Batist’s patient should have died nine years ago. Her pancreatic cancer failed to flinch in the face of the standard arsenal — surgery, radiation, chemotherapy — and Batist, an oncologist at McGill University in Montreal, Canada, estimated that she had one year to live. With treatment options dwindling, he enrolled her in a clinical trial of a hot new class of drugs called farnesyltransferase inhibitors. Animal tests had suggested that the drugs had the potential to defeat some of the deadliest cancers, and pharmaceutical firms were racing to be the first to bring such compounds to market.
But the drugs flopped in clinical trials. Companies abandoned the inhibitors — one of the biggest heartbreaks in cancer research over the past decade. For Batist’s patient, however, the drugs were anything but disappointing. Her tumours were resolved; now, a decade later, she remains cancer free. And Batist hopes that he may soon find out why.
The US National Cancer Institute (NCI) in Bethesda, Maryland, is recruiting stories, tissue samples and clinical data from up to 200 such ‘exceptional responders’ to learn why these patients benefited from drugs that failed most others. The effort is part of a larger push among cancer researchers to focus on single-subject, or ‘n-of-1’, studies that could offer new insights into the disease. The tactic initially met with resistance, says Charles Sawyers, a cancer researcher at the Memorial Sloan-Kettering Cancer Center in New York and an advocate of the approach. “It’s in vogue to talk about your n-of-1 study now,” he says. “But when I was in medical school this was called an anecdote — and it was a bad word.”
(via Boing Boing)