Characterizing Pediatric Brain Tumor Patient Derived Organoids (PDO) for Precision Drug Screening
Award: $100,000 (awarded 2025)
Principal Investigator: Carl Koschmann, MD, PhD, University of Michigan
Funding Partners: Catching Up With Jack
Despite major advances in tumor genomics, many children with aggressive or relapsed brain tumors still face treatment decisions guided by limited evidence. While molecular testing can identify tumor subtypes and mutations, it often cannot determine which therapies will actually work for an individual child. As a result, families and clinicians are left navigating trial-and-error approaches, exposing children to toxic treatments that may offer little benefit, particularly for high-risk tumors where time is critical and outcomes remain poor.
This project seeks to change that by advancing patient-derived tumor organoids (PDOs) as a powerful new precision medicine platform for pediatric brain tumors. PDOs are three-dimensional mini-tumors grown directly from a child’s own tumor tissue. Unlike traditional cell lines, which frequently fail to grow, take months to establish, or lose key biological features, PDOs preserve the tumor’s genetic makeup, brain-like microenvironment, and context all of which play a critical role in determining treatment response.
Building on a successful national collaboration with the Children’s Brain Tumor Network, this project will systematically validate PDOs across multiple pediatric brain tumor types, including diffuse midline glioma, medulloblastoma, ependymoma, and low-grade glioma. Using advanced molecular analyses, researchers will confirm that these organoids faithfully mirror the original tumors at the DNA, RNA, cellular and environmental aspects. Once validated, the PDOs will undergo rapid drug screening using FDA-approved and clinically relevant therapies, generating functional data on drug sensitivity within weeks of surgery.
By pairing deep molecular characterization with real-time drug testing, this work establishes a new framework for functional precision medicine in pediatric neuro-oncology, by testing therapies directly on a child’s own tumor model to inform future clinical trials and accelerate the identification of the most promising treatments for the most lethal childhood brain tumors.
