No Child Left Behind

Childhood cancer garners sympathy from all, but drug development from few.

He has a simple suggestion: grant an exclusive patent extension only if the pediatric clinical trial data are submitted within one year of approval in adults. “You can’t do it seven years later; you have to do it within one year,” he says. “I think if that happened, it would have a huge impact.”

While some researchers have hesitated to include children in early clinical trials, off-label use, he says, is “to my mind much more experimentation. What you get is everyone coming up with their own dose.”

When it comes to medicine, children are not pintsized adults. Their growing bodies and immature organs can tolerate some drugs at higher doses than adults, while some drugs reach toxicity at lower thresholds. Or, children might metabolize some chemotherapy treatments too rapidly, leaving them with all the side effects and few of the benefits.

To make clinical trials more efficient to conduct, some experts propose a change in structure that could make vital information easier and faster to obtain with fewer participants. The idea, described early this year in the Journal of Clinical Oncology, is known as the “rolling six” design, and it aims to get quicker answers once pediatric testing is under way. (A 2003 report to Congress noted that pediatric phase I safety trials typically don’t get started until two years after adult studies.) The framers of the rolling six idea predict it will be included in study protocols later this year.

And some initiatives are aimed at streamlining the process even before drugs reach the clinical trial stage. A program based at St. Jude Children’s Research Hospital in Memphis has, since 2004, been sifting through dozens of adult drugs looking for those that hold promise in children. Funded by the National Cancer Institute, the project identifies compounds that attack malignant cells in a way that might apply to childhood cancers. Once the compounds are flagged for testing, researchers begin looking for anti-tumor activity in mice.

There’s clearly an interest on the part of regulators in getting more [drug] development in children.

“We’ve been doing this for nearly 30 years at St. Jude on a much smaller scale,” says Peter Houghton, PhD, who heads the Pediatric Preclinical Testing Program. He and his colleagues in Memphis and several other sites can examine about a dozen drugs a year. “Our objective is to attempt to identify drugs that should be prioritized for pediatric cancers,” he says. In doing so, the investigators may also gain more insight into the biology of pediatric cancers.

Since the vast majority of oncology drugs are developed for cancers that don’t strike children, the program is a way to find drugs that might be otherwise overlooked in pediatrics. Preclinical testing has already had some positive findings, including favorable signs for an experimental class of compounds called insulin-like growth factor 1, or IGF-1, inhibitors. The hormone IGF-1, a cousin of insulin, is thought to have a role in many types of cancer. But among those that may be acutely dependent on IGF-1 is Ewing’s sarcoma—Callie Caylor’s cancer.

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