Opportunity and Other Grants
The Pediatric Brain Tumor Foundation's Opportunity and Other Grants fill critical needs in brain tumor research for children and teens.
Discussions with the PBTF's Research Advisory Network and the broader scientific community help us recognize candidates for funding through our Opportunity Grants program. The PBTF’s current Opportunity Grants focus on diffuse intrinsic pontine glioma.
COLLABORATIONS TO CURE DIPG
Although medical advances in the past 40 years have improved survival rates for many types of childhood cancer, DIPG is not one of them. This highly aggressive brain tumor defies treatment and currently has a zero-percent survival rate. Research on DIPG is performed in laboratories throughout the world, but previous efforts have been highly fragmented. The following Opportunity Grant awardees involve multi-investigator, multi-site efforts collaborating for a DIPG cure. These projects also hold tremendous potential for the study and treatment of other types of childhood brain tumors.
Project "DIPG All In"
Award: $100,000 over one year with the possibility of renewal
Principal Investigators: Kathy Warren, MD, Head of Neuro-Oncology Section, Senior Investigator in Pediatric Oncology Branch, National Cancer Institute, and Michelle Monje, MD, PhD, Stanford
Funding Partner: A Kids’ Brain Tumor Cure
When clinical testing of a drug begins, there can be significant gaps in knowledge or conflicting information about its basic biological performance. Securing additional funds to fill in these gaps is often challenging for labs; however, these gaps are important factors contributing to high drug failure rates in patients.
Project “All In” for DIPG is a novel public-private partnership led by the National Cancer Institute to coordinate DIPG studies and better qualify drug candidates for clinical trials in children by facilitating more thorough evaluation.
The project’s mission is two-fold:
- Establish and maintain a major effort to coordinate DIPG studies that will more efficiently and successfully advance DIPG therapies
- Establish a critical pipeline of therapeutic trials for all stages of the DIPG disease process.
The PBTF’s Opportunity grant supports All In’s “intramural project fund” at NCI. This precedent-setting grant is specifically designed to distribute funding as project requests are received and reviewed. The fluidity in available funding will greatly reduce researchers’ grant-writing time - shifting the concern from “What grant can I get?” to “What work needs to be done?”
This partnership also enables the PBTF to sit on the project’s Oversight Committee for evaluating funding requests. Being a part of it strengthens our expertise and insight into the research landscape at the national level.
"Project Open DIPG" Study Focused on HDAC Inhibitors
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: A Kids’ Brain Tumor Cure; CJR Memorial Foundation
This multi-institutional Project Open DIPG study uses state-of-the-art technology to determine the potency of candidate drugs belonging to the novel class of molecules called 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
Project Open DIPG is a joint venture by the Pacific Pediatric Neuro-Oncology Consortium, a previous PBTF Opportunity Grant awardee, and the Children's Brain Tumor Tissue Consortium.
The research builds on PNOC’s clinical trial portfolio through biospecimen collection, genomic sequencing, and an experimental animal model systems approach supported by CBTTC. The tissue samples will be added to CBTTC’s biorepository, and data from preclinical drug testing in DIPG tumor samples will be deposited into the CAVATICA platform.
The PBTF considers Opportunity Grant candidates on a continuous, case-by-case basis. Applications are accepted by invitation only; however, we welcome discussion of research ideas that fit with our strategic priorities, particularly if the ideas involve multi-institutional efforts.
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.
Brain Tumor Funders Collaborative's $3 Million Immunotherapy Initiative
The Brain Tumor Funders Collaborative is comprised of leading nonprofits and private foundations united to improve survival and quality of life for brain tumor patients. The BTFC funds multi-disciplinary teams and encourages proposals that span childhood through adult age groups.
The BTFC has pooled resources from six funders, including the PBTF, to invest in four immunotherapy projects that will help bridge the translational gap in brain cancer research. Each of these three-year projects will receive $750,000 grants from the BTFC.
The PBTF’s participation in this highly collaborative funding endeavor further solidifies the group’s dedication to advancing immunotherapy research for the benefit of children and adolescents diagnosed with a brain tumor – ensuring that pediatric brain tumors are a research priority and fostering productive collaborations between researchers studying brain tumors in adults and children.
It also presents the PBTF a remarkable and rare opportunity to multiply the impact of our $450,000 contribution to this large, strategic investment.
Learn more about our investment in the BTFC and the four selected projects here.
Novel Immune Mediated-Gene Therapy for Pediatric High-Grade Glioma
Award: $519,530 over three years
Principal Investigators: Maria G. Castro, PhD, R. C. Schneider Collegiate Professor of Neurosurgery, Professor of Cell & Developmental Biology at University of Michigan Medical School and Pedro R. Lowenstein, MD, PhD, Richard C. Schneider Collegiate Professor of Neurosurgery, Professor of Cell & Developmental Biology at University of Michigan Medical School
Co-Investigator: Karin Muraszko, MD, Chair and Julian T. Hoff, MD Professor, Neurological Surgery
Funding Partner: Samson Research Fund with funds raised through Think Fit for Kids Family Fitness Festival
Pediatric high-grade glioma (pHGG) is a common form of highly aggressive pediatric brain cancer that accounts for the leading cause of death by disease among children in the United States.
The University of Michigan Medical School’s Departments of Pediatrics and Neurosurgery have pioneered a treatment approach that uses gene therapy-mediated delivery of therapeutic genes into the tumor -- utilizing a combination of viral vectors that express: (1) a gene that induces tumor cells’ death and (2) another gene that trains the patient’s immune system to recognize and kill any remaining tumor cells. The viral vectors are delivered into the tumor cavity or the remaining tumor mass post-surgery to trigger an effective anti-tumor immune response.
This treatment strategy has been approved by the FDA for adult patients with glioblastoma multiforme, the most aggressive form of brain cancer, and a Phase I clinical trial has recently completed patient enrollment at the University of Michigan.
The Pediatric Brain Tumor Foundation’s grant will fund the needed experimental work in pre-clinical models to study the impact of the H3G34R mutation in reprogramming the glioma immune microenvironment and get FDA approval to implement this therapy in children.
Critical Mechanisms of Gene Regulation in 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
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.
The studies conducted over this project's two-year period will reveal the connection between and function of gene regulatory elements previously identified by Dr. Rivera’s lab as drivers of Group 3 medulloblastoma. This tumor subtype comprises 30% of all medulloblastoma cases and is associated with the poorest prognosis. Researchers will accomplish this by using new genomic and gene editing technologies to build 3D conformation maps of chromosomes/genomes from Group 3 medulloblastoma cells and directly test the function of key regulators of gene expression and their associated target genes.