Childhood cancer data on diagnosis, treatment, and outcomes are often disparate, but what if there was a publicly available, computational platform that facilitates prioritization and identification of pediatric drug targets?

On August 4, 2022, the NCI’s Childhood Cancer Data Initiative (CCDI) launched the Molecular Targets Platform. This knowledgebase allows researchers to browse and identify associations between molecular targets, diseases, and drugs and can be used for systematic identification and prioritization of the Food & Drug Administration’s (FDA’s) Relevant Molecular Target List (a spreadsheet that defines molecular targets relevant to the growth of pediatric cancer).

As an NCI instance of the Open Targets Platform, the Molecular Targets Platform allows comparisons of childhood data sets with tabular and graphic visualizations of genomic data. It facilitates the prioritization of targets by reducing the time spent monitoring and organizing scientific and medical literature. New childhood cancer data and additional functionality will be added to the Molecular Targets Platform as the project matures.

The Molecular Targets Platform builds upon the data and functionality of the Open Targets Platform while also including:

• the FDA Relevant Molecular Target Lists (FDA RMTL).
• analyses of pediatric oncology data sets from the Open Pediatric Cancer (OpenPedCan) project at the Children's Hospital of Philadelphia. Such data sets are made available by programs and initiatives like the following:
  • Therapeutically Applicable Research to Generate Effective Treatments (TARGET)
  • Open Pediatric Brain Tumor Atlas (OpenPBTA)
  • Gabriella Miller Kids First (Kids First) Neuroblastoma

This project is a collaborative effort between the Children’s Hospital of Philadelphia and The Frederick National Laboratory for Cancer Research, with input from NCI and FDA.

“This knowledgebase helps to validate FDA’s pediatric Relevant Molecular Targets List, thereby complimenting the Research to Accelerate Cures and Equity for Children Act and contributing to the important mandate for pediatric investigation by any industry partners developing drugs that target those variants,” shared Dr. Subhashini Jagu, a CCDI Data Platform Working Group member, and Mark Cunningham, technical project manager of Frederick National Laboratory.

Input from the data science community is highly valued to improve usability of this tool. Submit feedback to help improve usability as the CCDI continues to refine the platform. Please send your feedback and comments to ncichildhoodcancerdatainitiative@mail.nih.gov.