Funded Projects

Establishing and Credentialing a PLGA Xenograft Collection

This project addresses that challenge by establishing a credentialed repository of pediatric low-grade glioma patient-derived xenografts (PDXs), tumors grown in mice directly from a child’s tumor tissue. These models preserve the molecular, cellular, and architectural features of the original tumors and can be used for biologically relevant drug testing, biomarker discovery, and translational research. By building this shared infrastructure, the project lays the foundation for accelerating therapeutic discovery while reducing unnecessary risk to children. With support from the Pediatric Brain Tumor Foundation, this project has achieved substantial, measurable progress toward its original goals within its first two years, demonstrating both scientific rigor and strong return on philanthropic investment.

Award $375,000 over 3 years
Principal Investigator David H. Gutmann, MD, PhD, Washington University School of Medicine

Characterizing Pediatric Brain Tumor Patient Derived Organoids (PDO) for Precision Drug Screening

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.

Award $100,000
Principal Investigator Carl Koschmann, MD, PhD, University of Michigan
Funding Partners Catching Up With Jack

Investigating Mechanisms of Recurrence after Immune Gene Therapy in G34-Mutant Pediatric High-Grade Glioma

This project tackles that challenge head-on by developing two powerful, first-of-their-kind systems that allow researchers to watch, at single-cell resolution, how tumors respond to therapy, how resistance emerges, and which specific cells are responsible for recurrence. The team first built a highly faithful preclinical model of H3.3-G34–mutant glioma and tested an immune-stimulating gene therapy designed to both kill tumor cells and activate the child’s immune system. This showed that some tumors disappeared completely, others never responded, and some shrank only to grow back. This diversity mirrors what happens in children and creates an unprecedented opportunity to study the biology of success, failure, and relapse. In parallel, the researchers created a sophisticated genetic barcoding system, giving every tumor cell its own permanent “ID card.” This allows scientists to track which exact cells survive therapy, which ones expand afterward, and which rare clones drive recurrence. Together, these tools make it possible for the first time to pinpoint the specific tumor and immune-cell programs that determine whether a child responds to therapy or not. The approach bridges fundamental biology and translational research, opening the door to targeted strategies that eliminate resistant clones before they return.

Award $750,000 over 2 years (awarded 2024)
Principal Investigator Dr. Maria G. Castro, University of Michigan
Funding Partners Pediatric Cancer Foundation

BTFC 2 of 2: Global Alliance to Establish a CSF-based Liquid Biopsy Platform for Brain Tumors

This project seeks to transform that paradigm by developing a minimally invasive, CSF-based liquid biopsy platform using advanced DNA methylation profiling. Brain tumors shed DNA into the CSF, which can be safely collected through lumbar puncture. By applying a highly sensitive enzymatic methylation sequencing (EM-seq) approach, this platform can analyze tumor-specific DNA using extremely small amounts of CSF, thereby eliminating the need for surgical biopsy in many cases. To overcome the rarity of IGCTs and ensure scientific rigor, this initiative has formed a global alliance of more than 20 leading institutions across North America, Europe, Asia, and Australia. Together, they will assemble and analyze approximately 400 CSF samples, creating the largest and most comprehensive IGCT liquid biopsy dataset ever assembled. The result will be a validated diagnostic and monitoring tool with the potential to redefine how rare pediatric brain tumors are diagnosed and monitored over time.

Award $100,000
Principal Investigator Ching Lau, MD, PhD, Connecticut Children’s Medical Center
Funding Partners Brain Tumor Funders' Collaborative

BTFC 1 of 2: Maximizing Detection of Childhood Brain Tumors in Biofluids

This project seeks to change that. By applying groundbreaking deep whole-genome sequencing and advanced machine-learning analytics to more than 3,000 CSF samples collected from major St. Jude clinical trials, the team will dramatically enhance the sensitivity of ctDNA detection and provide an unprecedented view of how pediatric brain tumors evolve during treatment. Because CSF can be collected at diagnosis, throughout therapy, and at relapse, these liquid biopsies create a unique opportunity to track tumor behavior in real time and uncover the genetic mechanisms that drive resistance and recurrence. Together, these innovations will establish a new, clinically actionable framework for early relapse detection, more precise treatment monitoring, and the discovery of therapeutic targets. This work has the potential to fundamentally transform care for children facing the deadliest brain tumors moving the field closer to truly curative therapies.

Award $100,000
Principal Investigator Dr. Paul Northcott, St. Jude's Children's Research Hospital
Funding Partners Brain Tumor Funders' Collaborative

SNO: Neuro-Oncology Pediatrics Journal (2024-2025)

PBTF is honored to be the founding sponsor of Neuro-Oncology Pediatrics, a groundbreaking new journal dedicated exclusively to the science, care, and survivorship of children and AYA patients with brain and nervous system tumors. For families, clinicians, and researchers, this journal is far more than a publication. It is a lifeline.Pediatric neuro-oncology has been underrepresented in major scientific platforms. Discoveries were scattered, access was uneven, and critical insights were often hidden behind paywalls slowing progress for children who need it most. By ensuring this journal is open-access and globally accessible, PBTF is helping build a future where knowledge flows freely, collaboration grows stronger, and the best ideas reach the bedside faster.

Award $200,000 over 2 years
Principal Investigator Oxford Press

Accelerating the Clinical Translation of Nanomedicines for Pediatric Brain Tumors (ECD)

This project seeks to change that by developing a new way to deliver medicine directly and more effectively to pediatric brain tumors. This project entails designing tiny, highly specialized nanocarriers that can be injected into the fluid surrounding the brain and spine. These particles are engineered to bypass the brain’s protective barriers (BBB), travel deeper into tumor tissue, and release their medicine slowly over time. This approach has the potential to improve how long a drug stays active, reduce toxicity, and treat areas that conventional therapies cannot reach. Earlier versions of this strategy have shown success in adults, including a prior FDA-approved formulation, underscoring the promise of bringing a modernized, pediatric-focused version to children. By studying how different nanocarrier designs behave in the brain and how well they treat aggressive tumors in rigorous laboratory models, this work will generate the knowledge needed to advance the safest, most effective candidates toward clinical translation.

Award $300,000 over 3 years
Principal Investigator Joelle Straehla, Seattle Children's Hospital
Funding Partners Hearst Foundation (Year 1) and Christoper Brandle Joy of Life Foundation (Year 2 and 3)

Kindred - Elucidating the mechanisms of dissemination and therapy resistance in pediatric low-grade glioma

This project seeks to address that gap by uncovering why certain pLGG tumors spread and why they eventually stop responding to targeted therapies, even when those therapies initially show promise. Building on leadership of the world’s largest international disseminated pLGG consortium, encompassing more than 250 patients the research team has already demonstrated that dissemination is rarely driven by a single genetic mutation. Instead, early findings point to non-genetic factors and the tumor microenvironment, particularly changes in the CSF and immune signaling, as key drivers of aggressive behavior. To translate these insights into meaningful clinical impact, this project integrates four complementary approaches. First, researchers will analyze CSF samples using advanced proteomics, metabolomics, and single-cell sequencing to identify molecular changes that create a permissive environment for tumor spread. Second, they will study tumor samples collected before and after targeted MAPK-pathway therapies to uncover mechanisms of treatment resistance. Third, patient-derived cell lines and organoids will be used to test new combination therapies designed to overcome resistance. Finally, the development of the first in vivo mouse model of pLGG leptomeningeal dissemination, enabling direct testing of novel therapies and drug-delivery strategies.

Award $650,000 over 3 years
Principal Investigator Adrian Levine, MD, PhD, The Hospital for Sick Children (SickKids), Toronto
Funding Partners Team Syren, Kindred Foundation, Cancer Research Society

Advancing Pediatric Brain Tumor Research Through Crucial Infrastructure Support

The Children’s Brain Tumor Network (CBTN) is transforming pediatric brain tumor research by building the essential data, technology, and human infrastructure that powers precision medicine for children worldwide. With more than 8,000 participants enrolled and one of the world’s largest open, AI-ready ecosystems of clinical, genomic, molecular, and imaging data, CBTN is uniquely positioned to accelerate discoveries that no single institution could achieve alone. Its groundbreaking work, including leading the first ARPA-H award ever focused on pediatrics has already begun automating secure extraction of clinical data from electronic health records through the national RADIANT initiative, creating a scalable blueprint for real-time, multi-modal data integration across all tumor types.

Award $222,000
Principal Investigator Dr. Adam Resnick, Children's Brain Tumor Network

Novel Therapeutic Target for KIAA1549-BRAF-Rearranged Pediatric Gliomas

Pediatric low-grade gliomas (pLGGs) are the most common group of pediatric brain tumors and associated with significant morbidities. The most frequent driver alteration in pLGGs is KIAA1549-BRAF rearrangements. While MAPK pathway inhibitors have shown promise in targeting BRAF activation, they are not curative and many pediatric low-grade gliomas have primary resistance to these inhibitors. Through previous research funding, Drs. Pratiti (Mimi) Bandopadhayay's and Rameen Beroukhim’s research team at Dana-Farber Cancer Institute have identified a specific protein complex that is highly and specifically dependent on cells expressing KIAA1549-BRAF, with the potential to be a therapeutic target for KIAA1549-BRAF-rearranged pediatric gliomas. With this grant’s new funding, the team plans to test the protein complex’s impact on this type of tumor and investigate the combined treatment of MAPK inhibitors and complex suppression to determine if it results in sustained responses in pediatric glioma models.

Award $750,000 over 2 years
Principal Investigators Pratiti (Mimi) Bandopadhayay, MBBS PhD, and Rameen Beroukhim, MD PhD, Dana-Farber Cancer Institute
Funding Partners Catching Up With Jack; Hope for Kids Fund; Jacks Drive 55 Foundation through PBTF's PLGA Fund

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