Poster #26 - Aditya Lahiri

Towards a Unified Pediatric Cancer Resource: Integrating Clinical Trials, Molecular Targets, and Therapeutic Databases

Aditya Lahiri [1], Sangeeta Shukla [1], Eric Wafula [1], Kelsey Keith [1], Yuanchao Zhang [1], Dave Hill [1], Krutika S. Gaonkar [1,2,3], Run Jin [2,3], Komal S. Rathi [1,2], Yuankun Zhu [2,3], Bailey K. Farrow [2,3], Daniel P. Miller [2,3], Adam A. Kraya [2,3], Xiaoyan Huang [2,3], Bo Zhang [2,3], Zhuangzhuang Geng [2,3], Brian M. Ennis [2,3], Ryan J. Corbett [2,3], Nicholas Van Kuren [2,3], Matthew R. Lueder [2,3,4], Christopher Blackden [2,3], Saksham Phul [2,3], Miguel A. Brown [2,3], Alex Sickler [2,3], Asif T. Chinwalla [1], Alvin Farrel [1,5,6], Jo Lynne Rokita [1,2,3], Sharon J. Diskin [5,7], Adam C. Resnick [2,3], John M. Maris [5,6,7], Sarah K. Tasian [5,6,7], Deanne Taylor [1,7] [1] Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA; [2] Center for Data-Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA; [3] Division of Neurosurgery, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA; [4] Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA; [5] Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA; [6] Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA; [7] Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

Background: Pediatric cancers remain a leading cause of disease related mortality in children and adolescents. To advance therapeutic development, the RACE for Children Act expanded FDA requirements for evaluating oncology drugs in pediatric settings when the drug's molecular target is relevant to pediatric cancer. To support this mandate, the FDA maintains the Pediatric Molecular Target List (PMTL), which enumerates gene targets of pediatric relevance. Multiple public resources including PMTL, ClinicalTrials.gov, Open Targets, and the Illuminating the Druggable Genome (IDG) contain valuable but fragmented information on pediatric cancer therapies and targets. Integration of these resources has been hindered by inconsistent tumor nomenclature, particularly in ClinicalTrials.gov. Methods: Building on our CANTOS (Clinical Trials Automated Nomenclature and Tumor Ontology Standardization) framework, which benchmarked embedding based approaches for tumor name standardization, we extend this work to map tumor names from ClinicalTrials.gov, PMTL, Open Targets, and IDG into the International Classification of Diseases for Oncology (ICD-O). Using ICD-O as a unifying standard, we construct an integrated database that links tumors with molecular targets and drugs. Anticipated Results: This integrated framework will: 1. Identify drugs and targets associated with ICD-O standardized pediatric cancers from ClinicalTrials.gov. 2. Find common drug-targets for agents tested in registered clinical trials for pediatric and adult cancers. 3. For each FDA PMTL gene, summarize the associated pediatric cancers, adult cancers, and drugs. Conclusion: By standardizing tumor names across multiple resources and integrating ClinicalTrials.gov with PMTL, IDG, and Open Targets, this work creates a unified database for exploring therapies and molecular targets in pediatric cancer. Future work will extend this framework into a pediatric oncology knowledge graph, enabling complex queries, systematic comparisons between adult and pediatric cancers, and identification of regulatory or translational gaps. Such a graph will provide a scalable infrastructure to support pediatric cancer research and the goals of the RACE for Children Act.