The Molecular Targets Platform

The Molecular Targets Platform is a National Cancer Institute (NCI) instance of the Open Targets Platform with a focus on pediatric cancer data. This tool allows users to browse and identify associations between molecular targets, diseases, and drugs. The Molecular Targets Platform builds upon the data and functionality of the Open Targets Platform while also including:

• The FDA Pediatric Molecular Target Lists (FDA PMTL)
• Analyses of pediatric oncology datasets from the Open Pediatric Cancer (OpenPedCan) project at the Children's Hospital of Philadelphia:
  • Therapeutically Applicable Research to Generate Effective Treatments (TARGET)
  • Open Pediatric Brain Tumor Atlas (OpenPBTA)
  • Gabriella Miller Kids First (Kids First) Neuroblastoma

As the project matures, new pediatric cancer data and additional functionality will be added to the Molecular Targets Platform.

As the CCDI continues to refine the Molecular Targets Platform, input from the community is highly valued to help improve usability. Please send your feedback and comments to ncichildhoodcancerdatainitiative@mail.nih.gov .

The Open Targets Platform

The Open Targets Platform is the open-source infrastructure upon which the Molecular Targets Platform is built.

The Open Targets Platform is a comprehensive tool that supports systematic identification and prioritisation of potential therapeutic drug targets. By integrating publicly available datasets including data generated by the Open Targets consortium, the Platform builds and scores target-disease associations to assist in drug target identification and prioritisation. It also integrates relevant annotation information about targets, diseases, phenotypes, and drugs, as well as their most relevant relationships.

The Open Targets Platform is not an NCI program; their site is not affiliated with the NCI or any other US government agency. However, the Open Targets Platform is an open-source product, meaning that the data and code are shared freely with other organizations for development. The NCI has added pediatric cancer data and additional functionality into a separate instance of the Open Targets Platform under NCI control – the Molecular Targets Platform.

This About page will document all of the new data and features present in the Molecular Targets Platform. For detailed descriptions and tutorials of the built-in functions of the Open Targets Platform, please see their documentation or their most recent publication.

The Open Targets Platform undergoes regular updates to add new data and functionalities, which are integrated into the Molecular Targets Platform as soon as possible. To access Open Targets Platform data releases directly, please see their Data Downloads.

Pediatric Cancer Disease

Pediatric cancers are rare and heterogeneous, and have a different biology even from adult cancers of the same name. Due to the complexity and rarity, there was no international standard of classification until the end of 2021 when WHO updated their standards to include a distinct section for pediatric tumors. Considering the challenges and historical lack of standards, disease assignment and molecular subtyping is a challenging process. For Open Targets, classifying pediatric tumors starts with the pathologist’s report from original diagnosis followed by confirmation of the molecular features of the disease using the repository data. The specific molecular features examined for each disease were determined by the literature with expert review and curation from both bioinformaticians and clinicians. A final disease label is assigned based on the combination of the clinical pathology report and the molecular features in the data. If there is a discrepancy between clinical and molecular labels, samples are reviewed by a pathologist and final disease assignment is made in consultation with pathology, bioinformatics, and clinicians. For each disease, a non-exhaustive list of synonyms as well as the specific Experimental Factor Ontology (EFO) label used can be found on the individual page for each disease. For more details on disease assignment see OpenPedCan Molecular Subtyping and Pathology Documentation. For a summary table of the number of subjects included, a table is available for viewing and download.

Pediatric Cancer Data Sources

Pediatric Cancer Data Processing

Open Pediatric Cancer (OpenPedCan) project at the Children’s Hospital of Philadelphia, in partnership with the National Cancer Institute, is combining and harmonizing pediatric cancer datasets to accelerate pediatric cancer target identification and drug development. To read more about the OpenPedCan data processing methods, view the documentation.

Pediatric Cancer Data Visualizations