CHOP Leads Data Platform Development to Accelerate Novel Treatments for Childhood Cancers

The Molecular Targets Platform aids informed, data-driven decision making about childhood cancer drug development.

Childhood cancers represent the leading cause of death from disease in children over the age of 1. Comprising just 1% to 3% of cancers diagnosed annually in the United States, these cancers are considered rare. With data on diagnosis, treatment, and outcomes often stored at the institution where a child was treated, locating answers to scientific inquiries about childhood cancer is currently difficult, creating obstacles to improving outcomes for children with cancer through precision therapies.

But what if there was a tool useful for physician-scientists, advocacy groups, research communities, and pharmaceutical industry partners that encourages the development of treatment approaches for pediatric cancers? The Molecular Targets Platform aims to bring this vision to fruition.

Children's Hospital of Philadelphia has a leading role in the National Cancer Institute's (NCI) plan to develop this mechanism to streamline drug development for childhood cancers. As subcontractors of Leidos Biomedical Research, current operator of the Frederick National Laboratory for Cancer Research, John Maris, MD, and Deanne Taylor, PhD, are leading a team to facilitate the harmonization and usability of childhood cancer data by building this publicly available, computational platform.

"CHOP's strength in genomics, computational biology, and drug development present the ideal environment for this endeavor," said Dr. Maris, Giulio D'Angio Chair in Neuroblastoma Research at CHOP, and professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine.

Creating New Opportunities for Innovation

Recognizing the importance of including children in studies of applicable oncology drugs, the Food and Drug Administration authorized the Research to Accelerate Cures and Equity (RACE) for Children Act of 2017. Effective Aug. 18, 2021, RACE requires pediatric clinical trials for new oncology drugs with relevant molecular targets. Pharmaceutical companies must plan to study childhood cancers when investigating treatments for adults or obtain a waiver. The RACE Act dovetailed with a significant annual investment by the NCI's Childhood Cancer Data Initiative, creating new opportunities for innovation in developing therapies for childhood cancers.

To put the law into action, the FDA gathered experts to compile a pediatric molecular target list — essentially a catalog of the genes identified in childhood cancers. This data initially was housed in an Excel file that did not enable user-friendly cross-referencing of data about childhood cancers, diseases, and disease subsets.

Dr. Taylor, director of Bioinformatics in the Department of Biomedical and Health Informatics at CHOP and assistant professor of Pediatrics at the Penn Perelman School of Medicine, is leading the platform development after presenting early concepts of potential computational solutions to create a data repository. This "living" dynamic platform will be continuously built upon and reduce what previously constituted a few days' work for a computational expert to a few clicks by any user. The goal is for this to be the sandbox within which pediatric cancer experts, pharmaceutical industry leaders, FDA and NCI representatives, and patient advocates will interact to make informed data-driven decisions about childhood cancer drug development.

"Assembling a team across several different CHOP centers has provided a perfect conglomeration of capabilities necessary for this large-scale project," Dr. Taylor said. "Collaboration between the Department of Biomedical and Health Informatics, Center for Data Driven Discovery in Biomedicine and faculty in the Center for Childhood Cancer Research have provided a complimentary partnership to address a global challenge no one else is addressing: how to support and accelerate discovery of new cancer treatments for children."