Advancing Research
Funded Projects
Explore all of the Pediatric Brain Tumor Foundation’s currently and previously funded research awards. Search projects by keyword, brain tumor type, and more.
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Phase I/II trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ)
The development of MAP kinase-specific targeted inhibitors is the shifting therapeutic strategy for pediatric LGGs and offers many of these children new treatment options. Early phase investigations of RAF and MEK inhibitors in pediatric gliomas have demonstrated reasonable tolerability and superior responses in some patients compared to conventional chemotherapy.
- Award $350,000 over 2 years (2019 – 2021)
- Principal Investigators Dr. Arzu Onar-Thomas, St. Jude Children’s Research Hospital
Phase I/II study of MEK162 for children with progressive or recurrent low-grade gliomas and other central nervous system tumors
Phase I of this trial was rolled out in 2016 with support from PLGA Fund and had a target validation component to determine the ability of MEK162 to penetrate into the tumor as well as assess the ability of MEK162 to affect its target.
- Award $250,000 over 2 years (2020 – 2022)
- Principal Investigators Dr. Nathan Robison, Pediatric Neuro-Oncology Attending, Children’s Hospital Los Angeles, Dr. Mariella Gruber, Department of Pediatric Neuro-Oncology, Dana-Farber Cancer Institute, Dr. Karen Wright, Pediatric Neuro-Oncologist, Dana-Farber Cancer Institute
- Funding Partners Taylor Matthews Foundation, WhyNotMe? Foundation, Making Headway
Phase I/II and Target Validation Study of TAK-580 (MLN2480) for Children with Low-Grade Gliomas and Other RAS/RAF/MEK/ERK Pathway Activated Tumors
Oncogenic dysregulation of the RAS/RAF/MEK/ERK pathway has been described in a number of diverse malignancies, including low grade gliomas. Furthermore, mutations in BRAF, which occur in both monomeric and dimeric forms, drive the majority of pediatric LGG. Yet, only ten percent of pediatric LGGs possess BRAF V600E mutations, some of which are targetable by current type I inhibitors (dabrafenib, vemurafenib), which bind the activation site of the V600E mutation to prevent downstream signaling.
- Award $300,000 over 2 years
- Principal Investigators Dr. Karen Wright, Director of Neuro-Oncology, Assistant Professor-Pediatrics, Dana-Farber Cancer Institute, Dr. Daphne Haas-Kogan, Chair-Radiation Oncology, Dana-Farber Cancer Institute/Brigham and Women’s Hospital, and Sabine Mueller, Associate Professor of Oncology, University of California, San Francisco
- Funding Partners Thea’s Star of Hope, Starry Night Knoxville, Think Fit for Kids
COG ACNS 1833 Phase III Clinical Trial Comparing Functional and Quality of Life Outcomes after Selumetinib vs. Carboplatin/Vincristine in Children with Newly Diagnosed Low-Grade Glioma
In order to comprehensively compare CV and selumetinib treatment regimens, we will evaluate quality of life, cognitive, functional, and behavioral outcomes in children with newly diagnosed low-grade glioma.
- Award $400,000 over 2 years (2020 – 2022)
- Principal Investigators Dr. Peter de Blank, Cincinnati Children’s Hospital, Dr. Heather Conklin, St. Jude Children’s Research Hospital
"Project Open DIPG" Study Focused on HDAC Inhibitors
This grant enables researchers to: Compare the treatments’ effects on its own versus in combination with other types of treatments Study the cancer cells’ molecular functionality at the DNA and protein levels Determine animal model survival rates and duration
- Award $100,000 over one year with the possibility of renewal
- Principal Investigators Adam Resnick, PhD, Children’s Hospital of Philadelphia (CHOP), Javad Nazarian, PhD, Children’s National Health System, Benjamin Garcia, PhD, University of Pennsylvania
- Funding Partners CJR Memorial Foundation, PLGA Fund at PBTF
Critical Mechanisms of Gene Regulation in Medulloblastoma
Medulloblastoma is a complex form of pediatric brain cancer, with recent research uncovering an entire series of previously unknown subtypes. This grant awarded by A Kids’ Brain Tumor Cure, now part of the PBTF, will use pioneering technology to reveal innovative treatment approaches critically needed for children with medulloblastoma.
- Award $300,000 over two years
- Principal Investigators Miguel N. Rivera, MD, Assistant Professor of Pathology, Massachusetts General Hospital
- Funding Partner Christopher Brandle Joy of Life Foundation
Central Brain Tumor Registry of the United States Childhood and Adolescents Brain Tumor Report
Brain tumors are the most commonly diagnosed and deadliest form of childhood cancer in children and adolescents 0-19. Yet little is reported on these types of cancer. The Pediatric Brain Tumor Foundation funded a first-of-its-kind report to provide a statistically relevant and more accurate depiction of the incidence and impact of brain tumors in children and adolescents in the United States. The Central Brain Tumor Registry (CBTRUS) is the largest aggregator of cancer registry data on brain and other CNS tumors in the US and provides the most complete picture of population-based data for children and adolescents diagnosed with a brain tumor. Read the results of this report here.
- Award $64,000 over 1 year (2022)
- Principal Investigators Dr. Quinn Ostrom, Duke Medical Center; Carol Krutchko. Central Brain Tumor Registry of the United States (CBTRUS)
COG ACNS 1833 Phase III Clinical Trial Comparing Functional and Quality of Life Outcomes after Selumetinib vs. Carboplatin/Vincristine in Children with Newly Diagnosed Low-Grade Glioma
In order to comprehensively compare CV and selumetinib treatment regimens, this trial will evaluate quality of life, cognitive, functional, and behavioral outcomes in children with newly diagnosed low-grade glioma. Because overall survival for low-grade glioma is typically excellent, understanding the impact of treatment regimens on quality of life and function is essential.
- Award $400,000 over 2 years (2020 – 2022)
- Principal Investigators Dr. Peter de Blank, Cincinnati Children’s Hospital, Dr. Heather Conklin, St. Jude Children’s Research Hospital
