Clinical Trials in Era of Targeted Therapies

In Cancer, Research Not Separate from Clinical Care, Physician-Scientist Argues

December 17, 2010 | Bill Snyder

Cousins with melanoma caused by the same genetic mutation enroll in a randomized clinical trial of a promising experimental drug that targets the mutation.

Randomly assigned to different groups, one young man gets the new drug, experiences dramatic shrinkage of his tumors and is alive nine months later, while his cousin receives current therapy — the “control” — and is dead within two months.

The case, reported last fall in the New York Times, has raised questions in the scientific community about whether randomized trials are the best way to prove the effectiveness of new “targeted” cancer therapies.

An expert panel at Vanderbilt University Medical Center debated the issue Tuesday during a special Clinical Pharmacology Grand Rounds.

The panelists were Jeffrey Sosman, M.D., director of the Melanoma Program at the Vanderbilt-Ingram Cancer Center; Elizabeth Heitman, Ph.D., director of Research Ethics at the School of Medicine; and Alastair J.J. Wood, M.D., professor emeritus of Medicine and Pharmacology and a nationally known expert on drug safety.

Alastair J.J. Wood, nationally known expert on drug safety, was one of the panelists at Vanderbilt University Medical Center's Clinical Pharmacology Grand Rounds

None of the panelists suggested that randomized trials were outdated or passé. Indeed, Wood warned, “there’s a grave danger of embracing therapies based on their scientific rationale in the absence of robust clinical data.”

Part of the problem is that the public often confuses clinical research with clinical care, Heitman said. “If the patient benefits along the way, we like that, but that’s not the primary goal (of research),” she said.

From the audience, Carlos Arteaga, M.D., director of the Vanderbilt-Ingram Cancer Center’s Breast Cancer Program, challenged the implication that clinical research is not patient care.

He said his patients “know when they enroll in a clinical trial, the standard of care is there … At least in breast cancer, clinical research is patient care plus something else.”

Wood said the controversy might have been avoided if the study was “blinded,” so that neither the researchers nor the patients knew who got the experimental drug.

Sosman, principal investigator of a multi-center Phase II trial of the melanoma drug, agreed.

“I’m OK with the randomization,” he added, “because I will find (another) trial for the patients on chemotherapy who fail … My goal is to give the best clinical options to my patients, not to help (a drug company) get their drug approved.

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