# Pediatric Brain Tumor ConsortiumA multi-institutional consortium devoted to novel phase I and II clinical evaluations of experimental treatment approaches for pediatric CNS tumors.

> **NIH NIH UM1** · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · 2021 · $4,353,842

## Abstract

PBTC Project Summary/Abstract
The primary goal of the Pediatric Brain Tumor Consortium (PBTC) is to rapidly and effectively
contribute to the understanding and cure of pediatric central nervous system (CNS) tumors
through innovative, multidisciplinary and multi-institutional prospective clinical trials. PBTC clinical
trial development is not only based upon known prognostic factors but also upon an evolving
understanding of pediatric brain tumor biology. The PBTC will continue to utilize innovative study
designs and analyses based on state-of-the-art statistical science. Novel clinical trials will
incorporate relevant correlative pharmacology, biology, neuroimaging, immunologic and
genomics studies in an effort to define and integrate these modalities into a better understanding
of pediatric CNS tumors. These goals will be accomplished through a close and continued
partnership with the member institutions' basic and translational scientists as well as the clinical
trial experts. In addition, the PBTC welcomes partnerships with investigators outside the
consortium providing scientific, laboratory and regulatory support for innovative and high priority
clinical trials. The PBTC will continue to maintain and grow its relationship with the National
Cancer Institute (NCI) and its large network of adult and pediatric groups as well as with
pharmaceutical and biotechnology companies. One of the most important goals of the PBTC is to
successfully complete focused phase I and II clinical trials of novel agents and treatment
approaches that can be then be translated into large prospective phase II and III studies overseen
by the Children's Oncology Group (COG). The overarching long-term goal is to improve survival
outcome and quality of life for children with CNS tumor diagnoses that so often lead to high
morbidity and mortality such as diffuse intrinsic pontine gliomas, high-grade gliomas, infant
embryonal tumors, refractory medulloblastoma, ependymoma, and low-grade gliomas. To this
end, the PBTC will continue to focus upon introducing novel molecularly-targeted agents via
innovative trials to assess the presence of the target in tumor and surrogate tissue, evaluate the
ability of the agent to down-regulate its signaling target and better correlate pharmacokinetic and
pharmacodynamic endpoints with toxicities and clinical responses. The PBTC will also continue
to explore therapeutic opportunities for pediatric brain tumors through immunotherapeutic
interventions as well as via local delivery of treatments. By focusing on the most common solid
tumor in children which also accounts for the highest number of cancer-related deaths in
pediatrics, the PBTC will continue to support the NCI's mission of helping all people live longer,
healthier lives through its contributions to cancer research and identification of better treatments.

## Key facts

- **NIH application ID:** 10170275
- **Project number:** 5UM1CA081457-22
- **Recipient organization:** ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- **Principal Investigator:** Ira J Dunkel
- **Activity code:** UM1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $4,353,842
- **Award type:** 5
- **Project period:** 1999-04-01 → 2025-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10170275

## Citation

> US National Institutes of Health, RePORTER application 10170275, Pediatric Brain Tumor ConsortiumA multi-institutional consortium devoted to novel phase I and II clinical evaluations of experimental treatment approaches for pediatric CNS tumors. (5UM1CA081457-22). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10170275. Licensed CC0.

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