Pediatric Brain Tumors

NBTS funds, convenes, and project-manages research aimed at bringing better treatments to children with brain tumors — and at learning faster which treatments are worth advancing into clinical trials.

• National Brain Tumor Society (NBTS) played a role in helping secure the first and only U.S. Food and Drug Administration (FDA) drug approval for the treatment of recurrent H3K27M-mutant diffuse midline glioma (DMG) — a rare, aggressive brain tumor that predominantly affects children and young adults. In August 2025, the FDA granted accelerated approval to dordaviprone (Modeyso), formerly known as ONC201, which NBTS had previously supported through grant funding to the drug’s sponsor.

• NBTS’s flagship research initiative, the DNA Damage Response Consortium, brings together adult and pediatric researchers from nine major cancer centers to test different drugs and drug-device combinations for a new class of promising potential treatments in the laboratory, share data, and then bring the most promising investigational treatments forward to evaluate in clinical trials that match the right treatments with the right patients.
  
  • As part of this Consortium, the team at St. Jude Children’s Research Hospital is focusing on the toughest types of pediatric brain tumors, including diffuse intrinsic pontine glioma (DIPG)/diffuse midline glioma (DMG), atypical teratoid/rhabdoid tumors (AT/RT), and childhood ependymomas.
    
  • Since the Consortium launched in 2022, the team at St. Jude has received more than $1.2 million from NBTS for their work.
    
  • That investment has now produced a candidate treatment headed toward children. The St. Jude team studied a drug designed to make radiation therapy more effective by blocking a tumor’s ability to repair its own DNA — and found, unexpectedly, that the same drug also blocks a separate pathway that helps pediatric high-grade gliomas resist radiation. That laboratory work laid the foundation for an early-phase clinical trial in children with high-grade glioma. 

• Through the Collaborative Ependymoma Research Network (CERN), a program of NBTS, and in partnership with the Robert Connor Dawes Foundation, NBTS awarded the 2026 CERN & Robert Connor Dawes Scientific Fellowship to Siri Ippagunta, PhD, of the Mack Lab at St. Jude Children’s Research Hospital. Her two-year project targets XPO1, a protein that acts as a courier carrying messages out of a cell’s nucleus, and pairs that approach with other treatments that may work better in combination. The goal is to move these findings out of the laboratory and into a clinical trial for children with ependymoma. Dr. Ippagunta is the fifth CERN Ependymoma Fellow supported through this partnership.

• NBTS is currently funding the first-ever CERN PFA Ependymoma Translational Research Award, pursuing the cells that survive treatment in an aggressive infant tumor. An international team of renowned, interdisciplinary pediatric brain cancer experts, led by Johannes Gojo, MD, PhD, of the Medical University of Vienna, is engaged in a project titled “Targeting DNA damage response to eradicate ependymoma persister cells (DEEpend).” The group will receive $450,000 over three years.
  
  • PFA ependymoma is a rare but aggressive brain cancer that occurs in younger patients, primarily infants, with a mean age at diagnosis of three years old. These tumors are generally associated with a poor outcome, typically returning even after complete removal of the tumor and subsequent radiation and chemotherapy. There is currently no standard of care and no approved treatment for PFA ependymoma, and 10-year overall survival is only 56%. Our hope with this award is to accelerate scientific findings through Dr. Gojo’s translational research plan and to further stimulate additional and subsequent efforts to change the outcome for children diagnosed with this disease.
    
• NBTS has provided several years of funding, expertise, and advice to support the COllaborative Network for NEuro-oncology Clinical Trials (CONNECT) consortium. CONNECT is an international consortium focused on developing and testing novel therapies in early-phase clinical trials for children and adolescents with high-grade glioma brain tumors, including diffuse intrinsic pontine glioma (DIPG)/diffuse midline glioma (DMG). CONNECT includes 18 international partners designed to provide the most promising therapies to children with the poorest prognoses. Learnings from CONNECT are passed along to larger consortia to help speed up bigger trials.
  

NBTS connects the brain tumor community and provides resources to help better inform and prepare patients and their caregivers to access specialized treatment.

• The NBTS Personalized Support and Navigation Program — a service provided by an experienced team, including a highly skilled medical professional — ensures a patient and/or caregiver receives individualized attention, support, and information. This program has served families facing a pediatric brain tumor diagnosis.

• Similarly, NBTS offers a host of initiatives, such as Brain Tumor Support Conversations, Caregiver Support Conversations, and Grief Support Conversations, that help families impacted by a pediatric brain tumor diagnosis.

• Molecular testing increasingly determines which treatments and clinical trials a child may be eligible for. NBTS’s MyTumorID^® campaign helps patients and care partners understand biomarker testing, what the results can reveal about treatment options, and the questions worth asking a child’s care team.

• NBTS’s Clinical Trial Finder allows individuals to search for local clinical trials for specific tumor types, including clinical trial opportunities for pediatric patients.

• NBTS publishes a regularly updated report on newly opened brain tumor clinical trials across the United States, including studies enrolling pediatric, adolescent, and young adult patients.

• NBTS curates a list of resources available to help children, teens, young adults, and their families navigate the emotional and practical challenges that arise from a brain tumor diagnosis.

• NBTS also organizes events across the U.S. that bring the brain tumor community, including pediatric patients, together to honor, learn, develop meaningful relationships, and fuel momentum for our cause. Each September, NBTS marks Childhood Cancer Awareness Month with opportunities to take action on behalf of children with brain tumors.
  

NBTS and its volunteer advocates are working with members of Congress to advance policies and legislation that would provide better access to care and more funding for research for children and families facing a pediatric brain tumor diagnosis.

• With our champions in Congress, NBTS led efforts to introduce the Bolstering Research And Innovation Now (BRAIN) Act – now reintroduced in the 119th Congress as S.1330/H.R. 2767 – with growing bipartisan co-sponsorship. Most of this landmark legislation’s provisions are directly applicable to pediatric brain tumor research and care. For example, one proposed policy in the bill would create a new grant program to develop, study, or evaluate a model system for monitoring and care for adult, adolescent, and pediatric brain tumor survivors through their lifespan. This is particularly relevant for survivors of a pediatric brain tumor, as children often go on to experience a number of “late effects” from their treatment.

• In 2025, the National Cancer Institute (NCI) decided to end funding for the Pediatric Brain Tumor Consortium (PBTC) — for more than two decades, the only NCI-funded network devoted solely to early-phase brain tumor clinical trials for children and young adults — and move its work under the broader Pediatric Early-Phase Clinical Trials Network (PEP-CTN). NBTS disagreed with that decision and said so, first alongside the Pediatric Brain Tumor Foundation and the Society for Neuro-Oncology, and then in a fuller account of what was at stake for families. Despite an NBTS-led petition that collected more than 4,000 signatures, the decision stood, and now our focus is on ensuring a smooth transition that is responsive to the needs of the pediatric brain tumor community. NBTS led a sign-on letter urging that PEP-CTN receive the funding it needs to run a strong, viable pediatric brain tumor clinical trials program, and we continue to press the NCI and Congress to ensure the trial infrastructure and specialized expertise built over 20 years carries forward rather than dissipates.

• In February 2026, two NBTS-supported childhood cancer bills were signed into law. Congress passed, and the president signed, the Accelerating Kids’ Access to Care Act, which streamlines the enrollment process for eligible out-of-state providers under Medicaid and CHIP, and the Give Kids a Chance Act, which strengthens the study of new cancer drugs in children. NBTS advocated for both alongside partners in the childhood cancer community. Together they matter for families who travel across state lines to reach a pediatric neuro-oncology center, and for children whose future treatment options depend on new drugs being tested in kids rather than adults alone.

• NBTS is asking Congress to reintroduce and support the Medicaid for Every Child Act, which automatically enrolls all children in Medicaid from birth until age 18 without the need to re-enroll annually. This bill would ensure that no child would ever again be forced to grow up struggling to stay healthy due to a lack of health coverage.

• Sustained federal research funding. During the current federal budget (appropriations) cycle, NBTS is asking Congress to:

• Prioritize funding for brain tumor research within the Department of Defense’s (DOD) Congressionally Directed Medical Research Program (CDMRP).

• Increase funding for the DOD’s Peer-Reviewed Cancer Research Program (PRCRP) and continue to include pediatric brain tumors and brain cancer as eligible topics for funding.

• Fully fund the Childhood Cancer Data Initiative, establishing more efficient ways to share and use childhood cancer data.

• Fully fund the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act to guarantee that the programs inaugurated by the law are implemented and continue to make a difference for all those affected by childhood cancer, including pediatric brain tumors, now the leading cause of cancer-related death in children.

• NBTS has for years and continues to nominate pediatric brain tumor research advocates to serve on review committees for grants from the Peer-Reviewed Cancer Research Program of the Department of Defense, ensuring the pediatric brain tumor voice is represented at critical junctures of our nation’s biomedical research funding system.
  
• NBTS’s Chief Policy and Advocacy Officer currently serves as Co-Chair of the Alliance for Childhood Cancer’s public policy committee.