PA Hospital Launches Data Platform for Pediatric Cancer Drug Development

Children's Hospital of Philadelphia (CHOP) has announced the launch of a new analytics platform that will use pediatric cancer data to streamline and accelerate cancer drug development.

With funding from the National Cancer Institute (NCI) and a subcontract with Leidos Biomedical Research, CHOP researchers developed the Molecular Targets Platform to support pediatric research under the Research to Accelerate Cures and Equity (RACE) for Children Act.

RACE expands Food and Drug Administration requirements for sponsors of certain adult cancer drugs and products to assess the use of their medications in children, as long as there is a shared molecular target for the drug in both patient populations. The law is designed to facilitate pediatric research, which has sometimes been stalled by drug developers’ hesitance to test new treatments in pediatric populations, the press release notes.

To support the enactment of the law and encourage research, the FDA released a list of molecular targets in adult cancer that are considered relevant for pediatric cancer. However, data on pediatric cancer was dispersed in terms of representation of childhood cancer types. To address this issue, CHOP created the Molecular Targets Platform, the press release states.

"Those of us in the pediatric cancer research field were delighted when the RACE for Children Act passed, but for the legislation to truly have an impact, we knew we needed a computational ecosystem where all of these data could exist in a user-friendly interface," said Co-Principal Investigator John M. Maris, MD, the Giulio D'Angio chair in neuroblastoma research at CHOP, in the press release. "Through the hard work – and, importantly, the vision – of researchers in CHOP's Cancer Center, Department of Biomedical and Health Informatics, and Center for Data-Driven Discovery in Biomedicine, along with our collaborators at Leidos Biomed and the NCI, this platform will reduce the time it takes to make important data connections about childhood cancer from a few days or weeks to a few clicks of the mouse."

To build their platform, CHOP researchers pulled approaches from molecular medicine, computational science, and bioinformatics. They gathered data on pediatric cancer targets, genes, and pathways from six major data sources. This information was then integrated into a user-friendly interface.

The platform interface is publicly available and allows users to investigate various types of pediatric cancer data, including scored lists of cancer targets and profiles of a gene's relationship to other cancers and diseases. Though it is publicly available, the platform developers intend that it be used by researchers in academia, industry, and the FDA, and by patient advocates for research into childhood cancer therapies.

Other efforts to leverage data analytics to support pediatric cancer research are also underway.

The University of Chicago Pediatric Cancer Data Commons (PCDC) helped create data infrastructure for the Leukemia and Lymphoma Society’s LLS Pediatric Acute Leukemia (PedAL), a global pediatric cancer initiative, in 2020.

LLS PedAL was founded to accelerate precision medicine for pediatric cancer care. At the time of PCDC’s involvement, over 200 sites worldwide were set to participate in the initiative by testing targeted therapies for relapsed acute leukemia based on the specific genetic abnormalities driving the cancer. The research is ongoing, but gathered data from all sites will be consolidated into a single dataset.