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January 28, 2011 // By Holly Auer // Comments

Great Expectations: The Promise of Phase I Trials, Through Patients' Eyes

Cancer // Clinical Trials // Education // Health Care Quality and Safety // In the News // Research Share this article

A new study out this week from the bioethics think tank the Hastings Center is giving a voice to a group of patients that we don't hear much from -- the cancer patients who join Phase I clinical trials. For any given early trial, this group is small in number, but taken together, these patients represent the future of cancer therapies. Researchers are asking a lot of these patients: To hand themselves over to science -- and an often rigorous, time-consuming regimen of drug dosing, doctor's appointments, and constant monitoring -- at a time when may have just a scarce few months left to live. At bottom, joining a Phase I trial is an act of altruism, since these studies are designed only to test safe dosage levels and tease out side effects associated with the new drug -- not necessarily heal the patients who sign on. Though their own lives may not be prolonged by the early incarnations of the new drugs, these patients' role in research paves the way for bringing safe and effective new medicines to the masses who will later be diagnosed with the same diseases. Patients join these trials often at the end of a long road of failed therapies or when their disease is diagnosed too late for conventional treatment options. Often, their only other remaining option is to go on hospice care and prepare to die.

The new study shows that, despite going through a detailed informed consent process outlining the risks and (likely small) benefits of the trial, nearly 60 percent of Phase I and II trial participants who were surveyed said that they believed their cancer would be controlled by the study drugs. Fewer than 40 percent of them reported that they believed they would experience a "health problem" from the trial drugs. Those figures are both far out of line with typical early trial outcomes -- the researchers called it "unrealistic optimism." But is it wrong to dash their hopes that they'll be among the lucky ones who are, in fact, helped by the drugs? Or does outsized hope somehow help patients fare better?

Lynn Schuchter, chief of the Hematology/Oncology division in Penn's Abramson Cancer Center and an expert in care for metastatic melanoma -- a disease with one of the most grim prognoses in the cancer world -- shared her thoughts on the study in an MSNBC.com story published today. She said she worries that some patients whose expectations exceed the true therapeutic promise of their trial won’t opt for hospice care — and a better quality of life in their final months or weeks — because they haven’t admitted to themselves that they won’t get better. Still, said she was impressed at how well the patients in the new study actually understood the trial benefits and risks described to them. “They got the message,” she told MSNBC. “But at the end of the day, they were hopeful that they would be the patient who benefited from it.”

Usually, only about a quarter of patients involved in the early trials will see a benefit. When results exceed that, doctors, patients and their families rejoice. A new drug for metastatic melanoma that's nearing its long journey through clinical trials and federal regulation is one such example -- in its earliest trial, the majority of patients experienced tumor shrinkage and gained a median of seven months of life without disease progression.

That may not seem like a lot to average, healthy people, whose lives seem to spool out ahead of them for decades. But for patients like Chris Nelson, a Penn patient who lived for a year after being put on that drug when he was near death and was the subject of a New York Times series last year about the drug's development and testing, there's plenty of ways to make the time count:

At his wake, Mrs. Nelson told relatives she felt blessed that he had lived longer than expected. They had celebrated their 21st wedding anniversary. With the children, he had ridden every water ride at Six Flags Great Adventure.

“It’s a year I would never trade in,” she said.

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