Additional Pediatric Cancer Funding in the Offing?

In his State of the Union address earlier this month, President Donald Trump called for an additional $500 million to support pediatric cancer research over the next decade. Although the funding details are not yet clear, pediatric oncologists hope it will support research to identify new drug targets and develop therapeutics for cancers with known targets.

“I think that we are always very much in need of additional funding for childhood cancer research and very happy when there's an announcement such as this,” says Kimberly Stegmaier, MD, of Dana-Farber Cancer Institute and Boston Children's Hospital, both in Boston, MA. She notes that more than 80% of children with cancer are cured, yet it remains the leading cause of death from disease in children, and survivors must contend with long-term toxicities from therapies. “I do think there is a bit of this misperception that we're curing all childhood cancers and so we're done, but that's just frankly not true,” she says.

Peter Adamson, MD, of the Children's Hospital of Philadelphia (CHOP) in Pennsylvania, adds that although the pharmaceutical industry is the major funding source for research on adult cancers, it devotes few resources to pediatric cancers, which are relatively rare. “We're much more dependent on federal dollars and philanthropic research dollars than the large majority of the more common adult cancers, so certainly any potential increase in funding is very welcome news,” he says.

However, “the problem of childhood cancer is much larger than $50 million a year,” says John Maris, MD, also of CHOP, so there will be a continued need for federal grants and philanthropy. He adds that an unanswered question is whether the NCI will receive a new appropriation or will need to reallocate existing resources.

Maris recently participated in a teleconference with officials from the White House and the NCI, during which NCI Director Norman Sharpless, MD, emphasized data sharing as an area of unmet need within pediatric cancer research. Maris says that data-sharing efforts should be funded, but he would like to see more support for basic research and drug development.

“I think that this data-sharing coordination effort has to be partnered with discovery of the basic underpinnings of childhood cancers and how to take advantage of those basic underpinnings therapeutically,” he says. “We still have so much to learn about these developmental cancers in terms of their origins, their stunning heterogeneity, and their quick adaptation to the selective pressures of therapy.”

“One of our big challenges is that pediatric cancers generally have relatively quiet genomes in comparison to adults,” Stegmaier adds, making them less likely to respond to drugs that have effectively treated adult cancers.

To overcome this challenge, she thinks research should focus on developing therapeutics against known targets. For example, fusion oncogenes, expressed exclusively in cancer cells, drive tumor growth in a subset of brain cancers, leukemias, and sarcomas. Therapies that target mutations in chromatin regulators, which also underlie some pediatric cancers, could be pursued as well.

In addition, researchers want to unmask new therapeutic targets “to try to figure out what are the proper Achilles' heels in childhood cancers,” Stegmaier says. Other investigations might focus on immunotherapy and epigenetic-based treatments.

Adamson says the pace of scientific discovery is increasing, which will help lead to advances in treatment. “The scientific tools we now have at hand we couldn't have imagined 10 years ago, and so now is when you want to invest—when there's really a rapid acceleration in scientific understanding.” –Catherine Caruso