Gerald Batist, an oncologist at McGill University says that single-subject, or “n-of-1” cases are now more typical, and require further analysis: “If you talk to any oncologist, they’ll tell you about an unusual case or two like this. It’s time for us to stop just collecting anecdotes and dive deeper.”
Photo Credit: Claudio Calligris
Source: Nature News
An article, by Heidi Ledford, in Nature News says that medical oncologists might have more success with cancer therapies if they start relying on personal genomics; the thinking goes against prevailing ideas that one drug regime fits the needs of all cancer patients, or even of particular cancers such as colon or breast cancer. General clinical trials often don't tell the complete story, because they are general, statistical approach to predict viability.
Ledford writes about an exceptional case:
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.”
Since then, however, cancer-genome sequencing has forced researchers to reckon with the profound complexity of the disease. No two tumours are alike. Each has a web of mutations — typically numbering well into the thousands — that is as unique as a snowflake. Within these gnarled networks could lurk molecular signatures that reveal ways to target tumours, hidden hints for avoiding drug resistance and markers that could indicate which patients would respond to a given therapy. “This is a real treasure trove of data that has just been ignored,” says James Doroshow, deputy director for clinical and translational research at the NCI.This is indeed the future; and the very near future, to be precise, because it is both effective and prolongs lifespans. I can see why large pharmaceutical companies might not like this approach; they method is to conduct large clinical trails, with the aim of receiving regulatory approval, which means that the drug is both safe and generally effective for all classes of individuals, and not in particular cases only. It's a statistical approach, and a general regulatory approval is better for business.
And, yet I see a future where personal genome sequencing—not only a statistical approach— will become normative and large drug companies, unless they change, will become dispensers of general, everyday drugs such as pain killers and not become innovators. This might open a market for small, nimble biotech genomic companies that can quickly serve the immediate needs of both patients and doctors.
You can read the rest of the article in [Nature News]