Children’s Hospital of Philadelphia (CHOP) has helped launch a new computing platform that will harmonize pediatric cancer data, enabling researchers, pharmaceutical companies and advocacy groups to accelerate the pace of pediatric cancer drug development. With funding from the National Cancer Institute (NCI) through a subcontract with Leidos Biomedical Research, the current operator of the NCI’s Frederick National Laboratory for Cancer Research, CHOP researchers established the Molecular Targets Platform to facilitate pediatric research in response to the Research to Accelerate Cures and Equity (RACE) for Children Act, which requires companies to test pediatric cancer drugs used in adults when there is a common molecular target.
“Through this project, we are harnessing the power of integrated data to solve childhood cancer’s greatest challenges,” said co-principal investigator Deanne M. Taylor, PhD, director of Bioinformatics in the Division of Biomedical and Health Informatics at Children’s Hospital in Philadelphia and Assistant Professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine, leading the development of the new platform. “The Molecular Targets Platform will enable diverse communities to study new ways to understand and treat childhood cancer and will be an invaluable resource for drug discovery and development. This platform will also promote new hypotheses as humans leverage this computational ecosystem to make new discoveries.”
Childhood cancer research has long been thwarted by a reluctance among drug developers to test new treatments in children, in part because of the relatively small size of the affected population. The RACE for Children Act, passed in 2017 and passed in 2020, requires pharmaceutical companies to develop targeted cancer drugs for children if a drug with the same molecular target is tested in adults, even if the malignancy occurs in a different organ. For example, if a company is testing a targeted therapy for breast cancer and that genetic target is also relevant to a pediatric cancer, the company must also test the drug as a treatment for the pediatric cancer, unless it has a Food and Drug Administration (FDA) waiver. .
To facilitate the application of the law, the FDA has published a list of adult cancer molecular targets that are considered materially relevant for childhood cancer. However, there was no organized way to assess the list, as data on pediatric cancer genetics was scattered and uneven in representing the hundreds of childhood cancer types.
Through an NCI subcontract with Leidos Biomed and powered by the Childhood Cancer Data Initiative, CHOP researchers used their expertise in molecular medicine, computational approaches and bioinformatics to harmonize data from six key data sources related to childhood cancer targets, genes, and pathways. . The platform allows users to query multiple aspects of childhood cancer, from scored lists of cancer targets to profiles of a gene’s relationship to other cancers and diseases. The interface is publicly available for strategic research into childhood cancer therapies, with the intention of using it by researchers in academia and industry, as well as the FDA and patient advocates.
Those of us in the field of childhood cancer research were elated when the RACE for Children Act was passed, but for the legislation to really take effect, we knew we needed a computational ecosystem where all this data could exist in a user-friendly interface. Through the hard work – and most importantly, the vision – of researchers at 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 key data connections on childhood cancer from a few days or weeks to a few mouse clicks.”
John M. Maris, MD, co-principal investigator, Giulio D’Angio Chair in Neuroblastoma Research at the Children’s Hospital of Philadelphia and Professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine
The project is funded through a subcontract with Leidos Biomed, which provides over $3 million per year to CHOP through the NCI to develop and help maintain the new platform.
“We are grateful to those who recognized the need for this data platform and advocacy groups such as Kids v Cancer, who were instrumental in pushing for the approval of the RACE for Children Act,” said Dr. Maris. “With the Molecular Targets Platform, we hope we can accelerate the discovery of new and highly anticipated pediatric cancer treatments.”