Can cancer patients benefit from new drug trials?

NEW YORK (Reuters Health) - Patients with advanced head and neck cancer survived just as well on experimental drugs as they did on FDA-approved standard therapies in a new study.

These patients were part of phase I trials, an early step in the approval phase for a new drug and often the first time that drug is tested in humans. There has been controversy over whether advanced cancer patients - many of them desperate for any possible chance to get better - are getting taken advantage of in such trials, or whether they can really benefit from experimental drugs.

But the findings suggest that doctors should consider referring their terminally ill patients to such trials, the authors say.

“We expect that (the results) will increase enrollment of these patients in phase I clinical trials,” they write in the journal Clinical Cancer Research.

Dr. Ignacio Garrido-Laguna at The University of Texas M.D. Anderson Cancer Center in Houston and colleagues tracked 61 patients that had participated in phase I trials at their center over a five-year period. Fifty-nine of those patients had been on FDA-approved drugs before the start of the trials. The researchers calculated how long these patients had survived on their most recent treatment without their cancer getting worse. Then they made the same calculation of how well patients did on the experimental phase I drugs.

Patients survived an average of 12 weeks on FDA-approved treatments and 10.7 weeks on experimental drugs before their cancer progressed - outcomes that were not statistically different.

Four patients had some improvement in their cancer in response to the experimental drugs and 34 of them had no changes in their disease during drug trials. One patient died from a cause related to the treatment.

The point of phase I trials is to help researchers make sure that a drug is safe and to determine what the best dose is. Because these trials are not supposed to test whether the drug actually works - that comes later - “patients enrolled in them were rarely expected to derive benefit,” the authors write.

That may be changing as researchers get more tools to match patients with the right drug for their type of cancer, possibly improving the odds that they’ll benefit from the treatment, the authors say.

Still, some researchers and ethicists are wary of whether very sick patients are being given false hope.

“There are worries about whether people who are vulnerable in this way are able to give informed consent” to participate in a trial, Dr. Franklin Miller, a bioethicist at the National Institutes of Health in Bethesda, Maryland, who has written on phase I cancer trials, told Reuters Health. For researchers who are explaining drug trials to patients, “you have to be very careful,” he said.

There is also the question of whether you can really tell if someone benefited from being in a phase I trial, Miller said. Since these studies have no control group - a set of similar patients that isn’t getting the treatment - there’s no way of knowing if any positive outcomes were really a result of the drug, especially when the positive outcome being measured is how long someone’s disease was stable, he said.

But Miller thinks that the desire to join a phase I trial is a natural one for people who are dying of cancer. “They’ve exhausted all the (treatments) that are available and standard,” he said. If they want to keep fighting the cancer, he said, “there really isn’t any better option for them. I think this is genuinely what many people want to do.”

SOURCE: Clinical Cancer Research, online July 27, 2010.