Pediatric Brain Tumor Foundation
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of childhood brain tumors
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Funded Research Projects

The Pediatric Brain Tumor Foundation won't stop until there's a cure for every childhood brain tumor. Since 1991, the PBTF has supported projects at major research institutions around the world, fueling clinical trials, launching cutting-edge collaboratives and seeding the field for future discovery. The previously funded research projects listed on this page have helped lay the groundwork for today's and tomorrow's scientific discoveries and cures. Learn more about our current research funding programs here.


Early Career Development Grants

Resistance to BET-bromodomain inhibitors in MYC-amplified medulloblastoma
Principal investigator: Pratiti Bandopadhayay, MD, PhD, Dana-Farber Cancer Institute
Award: $300,000 over three years
Co-mentors: Rameen Beroukhim, MD, PhD and Charles Stiles, PhD, Dana-Farber Cancer Institute

Twenty-five percent of all medulloblastomas are driven by a gene called MYC. This gene makes the tumors behave aggressively and they are frequently resistant to the current treatments. Dr. Bandopadhayay and Dr. Beroukhim have recently shown that a new group of drugs called BET-bromodomain inhibitors are a promising novel strategy to treat these tumors. They have found that models of medulloblastoma in the laboratory are sensitive to a BET-bromodomain inhibitor, JQ1, which was developed by Dr. James Bradner at Dana-Farber Cancer Institute. However, experience with other novel agents have shown that cancers frequently evolve to become resistant. If the resistance mechanisms are understood, new drugs can be added to overcome resistance. The goals of the project are to characterize the resistance mechanisms to BET-bromodomain inhibition in MYC-amplified medulloblastoma. The hope is the results will guide development of therapeutic strategies, including use of combination therapies, to improve the efficacy of BET-bromodomain inhibition for the children with MYC-amplified medulloblastoma.

Unraveling medulloblastoma biology by proteomics
Principal investigator: Marc Remke, MD, Heinrich-Heine University, Dusseldorf, Germany
Award: $300,000 over three years
Mentor: Michael Taylor, MD, PhD, Hospital for Sick Children, Toronto, Canada
Funding Partner: Catching Up with Jack

This project will compare cancer cells and normal cells using tools designed to analyze DNA, RNA and proteins. To this effect, Dr. Remke will use mass spectrometry to profile the proteins of medulloblastoma, the most common malignant brain tumor in children. In addition to protein analysis, he will analyze the RNA of medulloblastoma by sequencing and the DNA by a microarray designed to look at methylation, which is like a switch for whether or not a gene will be turned into RNA. Dr. Remke will then integrate all the data from the protein, RNA and DNA analyses in the hopes that the results will help to inform clinicians about novel treatment strategies and to treat patients according to the aggressiveness of their disease.

Genetic susceptibility to ependymoma and interaction with perinatal risk factors
Principal investigator: Kyle Walsh, PhD, University of California, San Francisco
Award: $300,000 over three years
Mentor: William Weiss, MD, PhD, UCSF

Very little is known about what causes pediatric ependymomas. The most convincing factors identified to date suggest contributions from genetics and the immune system. By leveraging a large multiethnic patient population drawn from the California Birth Cohort, Dr. Walsh will compare the genomes of approximately 500 children with ependymoma to the genomes of 5,000 cancer-free children to identify genetic risk factors underlying this disease. Special focus will be given to genes involved in the immune response. He will also investigate interactions between genetic factors and potential perinatal risk factors, including birthweight, male sex and early life infections. Dr. Walsh believes that an enhanced understanding of the factors underlying pediatric ependymoma risk, including how genetic variation interacts with immune parameters, can change ependymoma research paradigms and usher in a new generation of studies that target the underlying causes of this disease.

Institute Grants

Research Projects

Epigenetic alterations define lethal CMP-positive ependymomas of infancy
Principal investigators: James T. Rutka, MD, PhD, FRCSC and Michael D. Taylor, MD, PhD
Institution: The Hospital for Sick Children, Toronto, Canada
Award: $1,000,000 over three years
Funding partner: Meagan's Walk Foundation

The principal investigators started a worldwide consortium, GENE (Global Ependymoma Network of Excellence) consisting of scientists, pathologists and clinicians from more than 30 centers around the world. Cumulatively, the members have contributed more than 400 PF-ependymomas with matched clinical data to the tumor bank in Toronto.

With funding from the PBTF, the PIs will continue to grow the tumor bank and develop biomarkers to discern PFA (poor prognosis) from PFB (better prognosis) ependymoma in a CLIA-certified manner. They will also develop additional human PFA xenograft models to study epigenetic agents for therapy and to study biology and response to novel agents in the relapse setting.

Development of a peptide vaccine for DIPG
Principal investigators: John Sampson, MD, PhD, MBA, Oren Becher, MD and Kendra Congdon, PhD
Institution: Duke University
Award: $400,000 over three years

Immunotherapy promises an exquisitely precise approach to treatment. However, it is limited in this role due to a lack of consistently expressed and tumor-specific antigens. Recently, a point mutation was discovered that provides a highly conserved and tumor-specific mutation, H3.3K27M, in 60 percent of DIPGs. Research objectives are to maximize immunogenicity of an H3.3K27M-containing peptide, to optimize vaccination timing in combination with radiation therapy in a murine model of H3.3K27M positive DIPG, and to perform IND-enabling studies.

A novel peptide vaccine targeting CMV antigens in recurrent medulloblastoma
Principal investigators: Eric Thompson, MD
Institution: Duke University
Award: $200,000 over two years
Funding Partner: Catching up with Jack

Recent findings have shown that cytomegalovirus (CMV) antigens are expressed in 92% of medulloblastoma cases. Targeting CMV antigens through peptide vaccination is therefore a candidate treatment strategy for this type of brain tumor. This novel strategy will be tested in a pilot clinical trial of a rationally-designed vaccine formulation containing multiple CMV peptides. The trial will enroll children with recurrent medulloblastoma and the primary goals are to determine safety and immunogenicity.

The oligodendrocyte developmental methylome to characterize progenitors for pediatric glioma
Principal investigators: Arturo Alvarez-Buylla, PhD and David Rowitch, MD, PhD
Institute: University of California, San Francisco
Award: $300,000 over three years

To better understand pediatric glioma origins, it is important to determine the precise stages of oligodendrocyte progenitor cell (OPC) development targeted by oncogenic mutations. The broad objective of this proposal is to use the methylome as a new index of oligodendrocyte developmental character and use this data to interrogate OPC-like character across different subtypes of human pediatric gliomas from brain stem and forebrain.

Personalized treatment strategies for DIPG
Principal investigators: Sabine Mueller, MD, PhD, Nalin Gupta, MD, PhD and Joseph Costello, PhD
Institution: University of California, San Francisco
Award: $300,000 over three years

Mutations in the gene encoding for histone H3.3 (K27M and G34V/R) have been reported as molecular drivers in pediatric HGGs. Moreover, the presence of K27M mutation correlates with worse clinical outcome. PIs will perform whole exome sequencing and gene expression profiling of tumor tissue from children newly diagnosed with DIPG to explore the genomic heterogeneity and to identify key alterations associated with treatment resistance and progression. This clinical feasibility study is being conducted through the Pacific Pediatric Neuro-Oncology Consortium.

Targeting tumor-associated inflammatory cells to ameliorate radiation-induced cognitive changes
Principal investigators: Nalin Gupta, MD, PhD and Susanna Rosi, PhD
Institution: University of California, San Francisco
Award: $150,000 over three years

Radiotherapy can cause late effects including progressive cognitive dysfunction. The activation of inflammatory pathways that occur along with tumor recurrence and after cranial irradiation produce many adverse effects, and no treatment is effective. The experiments proposed in this project will help provide a detailed understanding of macrophage accumulation in a rodent brain tumor model, and how modulating this response may affect cognitive performance.

Targeting Wnt-driven angiogenesis in pediatric glioma
Principal investigators: David Rowitch, MD, PhD and William Weiss, MD, PhD
Institution: University of California, San Francisco
Award: $150,000 over three years
Funding partner: Bryan's Dream Foundation

The Rowitch laboratory has shown that oligodendrocytes maintain a Wnt-activated program of angiogenesis at postnatal stages (Yuen et al., 2014, Cell in press). Moreover, many laboratories have shown conservation of oligodendrocyte-like features in human glioma. Therefore, PIs will test the hypothesis that gliomas have co-opted a mechanism for angiogenesis that is normally required during CNS development. This study may identify a novel target for anti-angiogenic therapy.

Core Resources

Biorepository, stem cell lines and xenografts
Principal investigators: Roger McLendon, MD and Stephen Keir, DrPH
Institution: Duke University
Award: $300,000 over three years
Funding partner: The Kyrie Foundation

Funding from the PBTF to the Biorepository Core provides for light microscopic and molecular analysis on all banked specimens in order that researchers have access to histologically and genotypically characterized tumor samples for diagnostic assessments in preclinical and clinical studies. In addition, funding supports ongoing development of pediatric brain tumor cell lines and patient-derived xenografts at Duke that enable the study of phenotypic and functional heterogeneity, the characterization of tumor subtypes, and preclinical drug testing. The resources of the core facilities are made available to researchers at Duke and other institutions.

Cell lines, animal models and tumor tissue bank
Principal investigators: Joanna Phillips, MD, PhD and Theodore Nicolaides, MD
Institution: University of California, San Francisco
Award: $100,000 over three years

Biospecimens obtained directly from the operating room during pediatric brain tumor resections are acquired, molecularly characterized and used to establish tumor cell lines and intracranial xenografts. These core resources, developed and maintained on an ongoing basis, are invaluable to help detect new therapeutic targets and to test novel therapies in animal models of the disease, guiding and improving future clinical therapies in pediatric brain tumor patients. The resources of the UCSF core facility are an essential component of the UCSF Brain Tumor Research Center. They are also made available to researchers at other institutions.

Opportunity & Other Grants

Rapid Molecular Testing of High-Risk Pediatric Brain Tumors
Award: $70,000 over one year
Principal Investigators: Jack Wadden, PhD, Carl Koschmann, MD
Funding Partner: Catching Up With Jack

Pediatric high-grade glioma (pHGG) and Diffuse Intrinsic Pontine Glioma (DIPG) are devastating childhood brain tumors with few patients surviving greater than 2 years. Over the last 2 decades the field has made great strides in uncovering the recurrent genetic drivers of these tumors through increasing use of molecular diagnostics (eg, DNA sequencing of tumor tissue). Sequencing can provide a more accurate diagnosis and prognosis and has become increasingly important for proper disease management and clinical trial enrollment for targeted therapies. Unfortunately, however, sequencing is far from optimized for clinical utility as turnaround time for tumor sequencing is typically 2 - 4 weeks and rarely available prior to radiotherapy or trial enrollment for agents given concurrently with radiotherapy. Furthermore, due to the risk of morbidity/mortality from repeat biopsy to track tumor mutations, it is not safe or feasible. To address these issues, this study takes an interdisciplinary approach to leverage a highly sensitive amplification technique currently being used in rapid COVID-19 diagnostics (LAMP) to enable rapid, low cost and low complexity detection of tumor mutations directly from the patient CSF and plasma. The outcome, if successful, will enable clinicians to more accurately and rapidly diagnose specific tumor mutations in order to recommend customized treatment options for the child’s specific tumor at the time of diagnosis and throughout the patient’s tumor journey, with little or no increased risk from complications due to invasive biopsies.

Pacific Pediatric Neuro-Oncology Consortium, Operations Center
Project leaders: Michael Prados, MD, and Sabine Mueller, MD, PhD, University of California, San Francisco
Award: $500,000 over two years with the possibility of renewal for a third year
Funding Partner: Vs. Cancer Foundation

The Pacific Pediatric Neuro-Oncology Consortium, or PNOC, is a network of children’s hospitals formed to provide children with brain tumors access to clinical trials of innovative treatments. The mission of the PNOC is to pursue clinical strategies that capitalize on each patient’s tumor-specific molecular and genetic make-up.

Each institution in the network has dedicated pediatric neuro-oncology research laboratories that lay the necessary groundwork to move quickly towards implementation of novel clinical trial concepts based on preclinical research. Each of the 15 participating hospitals also has the requisite multidisciplinary team of brain tumor specialists for optimal patient care and in-depth understanding of the needs of families and children suffering from this disease.

The Operations Center coordinates the activities of the PNOC across all participating centers and clinical trial protocols and is an essential component for the overall success of PNOC.

Immunosignature Strategy for Development of Clinical Biomarkers and Identification of New Drug Target Candidates for Pediatric Brain Cancer
Project leaders: Bob Carter, MD, PhD, University of California, San Diego; Stephen Albert Johnston, PhD, Arizona State University; and Robert Wechsler-Reya, PhD, Sanford Burnham Prebys Medical Discovery Institute
Award: $234,000
Cofunder: ABC2 (Accelerate Brain Cancer Cure)

Antibody-based profiling shows promise as a general approach to diagnosis and as an indicator/biomarker of response to treatment. Researchers have developed a novel technology platform to detect patterns of tumor-recognizing antibodies, or immunosignatures, that differ by tumor type and disease state. 

Using this platform, researchers will assay human tumor and blood samples to define signatures that correlate with each of the four molecular subtypes of medulloblastoma. Simple blood tests analyzing these immunosignatures patterns have already been shown by Dr. Johnston's lab to recognize different types of adult brain cancer.

In specialized laboratory models of medulloblastoma, researchers will test the utility of the immunosignatures as dynamic biomarkers for early detection of tumor growth/relapse and of tumor responsiveness to drugs. The tumor-associated proteins that elicit the immunosignatures will also be identified, thereby uncovering candidate targets for novel therapies for medulloblastoma.

Project:EveryChild (The Children’s Oncology Group)
Project leader: Peter Adamson, MD, Chair, Children's Oncology Group, Alan R. Cohen Endowed Chair in Pediatrics at the Children's Hospital of Philadelphia

The Children’s Oncology Group (COG) unites more than 9,000 experts in childhood cancer at more than 200 leading children’s hospitals, universities and cancer centers across North America and other continents in the fight against childhood cancer.

The COG’s Project:EveryChild is an initiative that will create a fundamental platform for translational research. The initiative involves taking extra tissue available from children who must undergo a diagnostic procedure and then storing samples in COG’s state-of-the-art biorepository which serves as a worldwide resource. Laboratories studying these tumor tissue samples can then link clinical data from each child, such as the treatment the child received and how effective it was, as their research moves forward. Project:EveryChild will result in a well annotated childhood cancer biobank to enable robust discovery research and a platform for personalized medicine.

The study is open to all children diagnosed with cancer. Funding from the PBTF specifically supports the acquisition of follow-up clinical information and central nervous system (CNS) tumor biospecimens. The costs of obtaining the biospecimens and corresponding clinical follow-up data obtained at diagnosis or relapse from children with brain tumors are often not covered by grants from the National Cancer Institute.

BRD4 as a Therapeutic Target for Medulloblastoma
Project leader:
Rameen Beroukhim, MD, PhD, Dana-Farber Cancer Institute
Funding Partner: Christopher Brandle Joy of Life Foundation

In approximately 25 percent of medulloblastoma cases, tumor growth is driven by the amplification of a gene called MYC. The majority of children with MYC-amplified medulloblastoma die of their disease. Other pediatric cancers that harbor amplifications of MYC family members, notably neuroblastoma, are also similarly associated with devastating outcomes. Novel therapeutic strategies are desperately needed in the clinic for children with these cancers. Experience with novel agents, however, has shown that cancers frequently evolve to become resistant. A promising therapeutic approach is to develop combination treatments that include drugs to overcome or circumvent the development of resistance. In laboratory studies, Dr. Beroukhim has shown that MYC- amplified medulloblastomas are sensitive to a class of chromatin modifiers called BET-bromodomain inhibitors. The specific aims of his proposal are to fully elucidate the mechanism of action of this family of inhibitors and thereby gain insight into the genes and proteins that confer resistance. His results will guide strategies to optimize the efficacy of BET-bromodomain inhibitors for children with MYC-amplified medulloblastoma and provide a framework to study cancer's evolution in response to this novel class of cancer drugs.


  • Baylor University-Texas Children's Hospital
  • Barrow Neurological Institute
  • Boston Children’s Hospital
  • Children’s Hospital Los Angeles
  • Children's Hospital of Philadelphia
  • Children's Research Institute
  • Children's National Medical Center
  • City College of New York
  • Columbus Children's Hospital Research Center
  • Cornell University
  • Dana-Farber Cancer Institute
  • Duke University Medical Center
  • Evanston Hospital
  • Emory University School of Medicine
  • Harvard Medical School Dana-Farber Cancer Institute
  • Heinrich Heine University, Dusseldorf, Germany
  • Henry Ford Health System
  • Hospital for Sick Children, Canada
  • Howard Hughes Medical Institute
  • Johns Hopkins University Oncology Center
  • Ludwig Institute for Cancer Research
  • Massachusetts General Hospital
  • Mayo Clinic Cancer Research
  • Memorial Sloan-Kettering Cancer Center
  • Murdoch Children’s Research Institute, Australia
  • New York University Cancer Center
  • Pediatric Brain Tumor Consortium
  • Royal Children's Hospital, Australia
  • Scripps Research Institute
  • St. Jude Children's Research Hospital
  • Stanford University
  • University of Alabama at Birmingham
  • University of California, San Francisco
  • University of Cincinnati
  • University of Colorado Health Sciences
  • University of Iowa Cancer Research Center
  • University of Michigan Medical Center
  • University of Pittsburgh
  • University of Texas M.D. Anderson Cancer Center
  • University of Utah
  • University of Virginia
  • University of Washington Cancer Center
  • Washington University, St. Louis
  • Women's and Children's Hospital Northwestern University


The PBTF sponsors scientific meetings where the latest developments are discussed and new collaborative research efforts are often forged to advance the search for the cause of and cure for childhood brain tumors. These have included:

  • American Association of Neurological Surgeons/Congress of Neurological Surgeons Section on Tumors (AANS/CNS) meeting
  • American Association of Pediatric Oncology Social Workers, Education Day for new pediatric oncology social workers, annual meeting
  • Brain Tumor Epidemiology Consortium meeting
  • International Conference on Brain Tumor Research and Therapy
  • International Symposium on Pediatric Neuro-Oncology
  • Neurobiology of Disease in Children, Brain Tumor Symposium
  • Society of Neuro-Oncology Annual Meeting
  • SNO’s Pediatric Neuro-Oncology Basic and Translational Research Conference (PNO-BTRC)

In addition, the PBTF provides funding to the Central Brain Tumor Registry of the United States, which works with population-based registries to track all primary brain and central nervous system tumors diagnosed in the United States. The data are published in an annual statistical report.

A PBTF grant also established and supported the annual Peter A. Steck memorial lecture and recognition award for young investigators. The lectureships were delivered by world-renowned experts at Harvard Medical School, the Ludwig Institute, MD Anderson National Cancer Institute, Salk Institute, Scripps Research Institute, St. Jude Children’s Research Hospital, the University of California at San Francisco, University of Texas Southwestern Medical Center, and the University of Toronto. Recipients of a recognition award have included young investigators at Baylor College of Medicine, Cold Spring Harbor Laboratories, Duke, MD Anderson, Stanford University, St. Jude Children’s Research Hospital, University of Pennsylvania, and the University of Virginia.