An estimated 2,000 children die of cancer each year, and the overall incidence of childhood cancer has been slowly increasing since 1975. Despite significant advances against certain pediatric cancers, including acute lymphoblastic leukemia, there are still some types of cancer for which there are few or no effective treatments. As John London found out, new drug development in the field is slow, often lagging way behind adult treatments, and few compounds are designed specifically for children. “I was on my own, as many parents are,” London says. “The medical community had no interest.”

That is in large part due to a practical reason: Childhood cancers make up less than 1 percent of all cancers diagnosed each year, according to the American Cancer Society. That 1 percent is not much of a market for drugmakers, who rack up an estimated $1.4 billion in out-of-pocket costs while bringing a novel drug to market. They would never recoup that treating the 700 children diagnosed with neuroblastoma annually, or the 100 diagnosed with diffuse intrinsic pontine glioma, a deadly brain tumor.

“The big elephant in the room is the cost of this type of research,” says Raphaël Rousseau, director of pediatric oncology drug development at pharmaceutical giant Roche. Combined with the small potential market, that’s led very few pharmaceutical companies to invest in developing drugs for pediatric cancer. Merck has one ongoing pediatric oncology trial. Pfizer is testing preclinical therapies only. Novartis leads the pack, with seven drugs in clinical trials for children’s cancer.

Where Big Pharma is absent, government has stepped in. Most pediatric clinical trials are operated by the National Cancer Institute’s (NCI) Children’s Oncology Group (COG), which runs approximately 40 to 50 therapeutic trials across the country at any one time, according to Peter Adamson, chairman of the organization and a pediatric oncologist at the Children’s Hospital of Philadelphia. Yet even with federal funding, pediatric cancer research receives only a fraction of the money that adult cancer research gets, and it’s decreasing. In 2013, the NCI invested $185.1 million from a $4.79 billion budget in pediatric cancer research, the lowest amount since 2009.

“The options we have now to be explored are really blossoming, but the funds available to do the studies that need to be done are shrinking,” says Richard O’Reilly, chairman of pediatric oncology at Memorial Sloan Kettering Cancer Center, in Manhattan. ‘We don’t want future generations to look back on this time and ask, ‘What the hell were they doing?’”

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