Pediatric Brain Tumors

• 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 for 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’s has received $2 million for their work from NBTS.
    
• 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 is currently funding the first-ever CERN PFA Ependymoma Translational Research Award.  An international team of renowned, interdisciplinary pediatric brain cancer experts, led by Dr. 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).” With the overall aim of translating promising research into the clinic for a particularly aggressive type of pediatric brain tumor, 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 3 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 percent.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 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 and Grief Support Conversations, that help families impacted by a pediatric brain tumor diagnosis.
  
• 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 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.
  

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 get the Bolstering Research and Innovation Now (BRAIN) Act introduced in Congress in July 2024. Most of this landmark piece of bipartisan 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. 
  
• NBTS is advocating for the Accelerating Kid’s Access to Care Act (AKACA), which continues gaining sponsorship and support in Congress. This bill would streamline the enrollment process for eligible out-of-state providers under Medicaid and CHIP programs. In July, NBTS collaborated with the Pediatric Brain Tumor Foundation and the Leukemia Lymphoma Society on a town hall with Senator Grassley (R-IA) to help advance this legislation.
  
• 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.
  
• NBTS joined with other groups to again successfully advocate for a resolution designating an official DIPG Awareness Day in the United States in 2025.
  
• During the current federal budget (appropriations) cycle, NBTS is asking Congress to provide:
  
  • $150 million for the Peer-Reviewed Cancer Research Program (PRCRP) at the Department of Defense (DOD), and include brain cancer and pediatric brain tumors as topics eligible for funding.
    
  • Full funding ($50 million) to continue the Childhood Cancer Data Initiative, establishing more efficient ways to share and use childhood cancer data.
    
  • Full funding ($30 million) for 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 is asking Congress to support the Give Kids A Chance Act of 2025 (H.R.1262, S.932) to unlock potential new treatments for kids. The National Brain Tumor Society has endorsed this bill alongside member organizations in the Alliance for Childhood Cancer. This legislation also include:
  
  • Creating Hope Reauthorization Act, which would enable the use of priority review vouchers as economic incentives for pediatric disease treatment developers to utilize as an expedited FDA review pathway.  
    
• NBTS also ensures the pediatric brain tumor voice is represented at the highest levels and most critical junctures of our nation’s biomedical research funding system:
  
  • NBTS’s CEO sits on the Childhood Cancer Data Initiative’s Steering Committee.
    
  • 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.