Scenes from a cancer trial

By Jeremy Manier

Neuroblastoma patients recently got some of the best research news they’ve had in a while – but the news wasn’t perfect for everyone.

Word of the advance came in an announcement last month from the national Children’s Oncology Group. A Phase III trial of an antibody-based treatment for neuroblastoma (protocol ANBL0032) had found that the treatment significantly increased patients’ chances of survival. The trial was immediately halted – standard procedure in cases where a trial is so successful that it would be unethical to deny patients the treatment.

The antibody treatment yielded a striking survival gain of about 20 percent for children with the deadly form of cancer. Neuroblastoma researchers such as Susan Cohn, M.D., were elated.

“This is as successful a story as we’ve had in neuroblastoma treatment in many, many years,” Cohn told me in a recent interview. “We hope to be able to incorporate this as part of our standard approach.”

The protocol is on hold to study toxicity issues that arose in some patients, but experts hope the treatment will be available soon for more patients. The March 19 statement from COG concluded, “We now expect that this immunotherapy may eventually become a standard part of high-risk neuroblastoma treatment after stem-cell transplant.”

So what’s the downside? It’s simply the nature of such clinical trials, in which not everyone can get the superior treatment.

Patients are randomized at the start of a trial like this one; some get the new treatment, some get the standard, established protocol. Some families opt out altogether. No one knows at the outset whether the new protocol will be better, the same or perhaps worse than the existing treatment.

But it’s clear now that in this case, patients who got the new protocol stood a markedly better chance of surviving their cancer than those who stayed with the old form of treatment. A 20 percent improvement is no guarantee, of course; some patients on the new protocol died, and many who got the existing treatment did fine. Yet an outcome like this can raise a tantalizing “what-if” question in the minds of some parents. Might a child have had a better chance at survival if randomization had placed him or her in the new treatment group? What about parents who may feel guilt over not taking part in a trial that could have improved their child’s outcome?

Such doubts may be impossible to resolve. It’s difficult to imagine replacing randomized trials as the gold standard for gathering¬†scientific evidence that a treatment works.

“You just never know until you do the study,” Cohn said. “We are so grateful to the families who are willing to participate in these trials.”

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