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Childhood Cancer Data Initiative (CCDI): CCDI Pediatric In Vivo Testing Program - Orthotopic CNS Tumors

The goal of this project is to advance the development of effective therapies for pediatric brain cancers through preclinical in vivo testing of novel agents. We utilize a well-characterized panel of approximately 150 patient-derived orthotopic xenograft (PDOX) models that replicate the histopathologic and molecular features of the original patient tumors. These models represent multiple pediatric CNS tumor types, including high-grade glioma, medulloblastoma, ependymoma, diffuse intrinsic pontine glioma (DIPG), and atypical teratoid/rhabdoid tumor (ATRT), across diverse disease stages and molecular subtypes.

As part of this effort, the PDOX models are being molecularly characterized to identify promising therapeutic candidates. Molecular profiling includes whole exome sequencing (WES), RNA-seq, MethylEPIC array, CytoSNP array, and DNA fingerprinting for quality control. Demographic and clinical data (e.g., diagnosis, disease site, disease status) are also shared.