For children with cancer, the best treatment most often is available through participation in clinical trials, according to a childhood cancer expert who spoke on the topic at the American Academy of Pediatrics' annual meeting, held recently in San Francisco.

"Children in clinical trials have better outcomes than those not participating in clinical trials," noted Sharon B. Murphy, MD, professor of pediatrics at Northwestern University Medical School and chief of the division of hematology/oncology at Children's Memorial Hospital in Chicago.

Murphy said the tendency of patients in trials to do better than others — even if they are not among the group getting the experimental treatment — is known as the "inclusion benefit." One reason to expect it among kids is that cancer in children is so rare that it is best treated by experts.

Those experts are found almost exclusively in childhood cancer centers, where they spend a large portion of their time planning and working in the clinical trials. They hope these trials will improve children's survival as much in the future as they have in the past, said Murphy.

The trial design team also includes nurses and pharmacists who are experts in childhood cancer. Pathologists and other experts on the hospital's tumor board review the children's original diagnostic materials, to make sure the original diagnosis is correct.

Then all aspects of the trial are reviewed by the hospital's Institutional Review Board (IRB), and the trial is allowed to begin accepting patients only if it passes the IRB's scrutiny.

"By the time the trial is approved, it's like having numerous built-in expert second opinions right there, helping you, so it's no surprise the outcome's better," noted Murphy.

A childhood cancer expert with the American Cancer Society (ACS) agreed that clinical trials are the best option for children with cancer.

Years ago, most children were treated after surgery with both radiation and chemotherapy. But early trials showed the radiation usually wasn't needed if the surgery was successful, noted Teresa J. Vietti, a pediatric cancer specialist at the Washington University School of Medicine in St. Louis, and a member of the ACS editorial board.

"Now the best treatment is almost always multiple drugs — and what we're trying to do in clinical trials is find out if there's a more effective or less toxic combination than the one we're giving now," said Vietti.

Usually, she noted, that means both groups in a trial get the standard treatment, with an addition or substitution of one drug in one group.

Vietti explained that all trials protect patients by having data monitoring boards (DMB), independent of those running the trials, to oversee the trial.

In a clinical trial for T-cell leukemia, the DMB saw during the trial that the experimental treatment was so much better than the standard one that the trial was stopped and all patients put on the experimental treatment, Vietti noted.

But the DMB may stop the trial if the new treatment isn't working.

"We're not going to continue to treat children with an inferior drug," she said.

And a parent may withdraw a child from a trial at any point, so the child is protected in multiple ways, from design through completion of the trial.

Vietti noted that parents and children participating in clinical trials get the added bonus of knowing they are contributing to progress against childhood cancer.

"All children with cancer should be entered in a clinical trial if they are eligible, because it provides the best treatment, and the best oversight, as well as the best hope for further progress," said Vietti.