Pediatric Brain Tumors

• NBTS launched the DNA Damage Response Consortium, in partnership with Yale Cancer Center, in early 2022. 
  • The 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 will focus 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. 
• 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.
• With support from the CERN Foundation, a program of the National Brain Tumor Society, Dr. Sriram Venneti (University of Michigan) and his lab discovered in 2021 that the common diabetes drug metformin suppressed tumor growth in group A posterior fossa ependymoma laboratory models. 
• NBTS awarded the 2022 CERN and Robert Connor Dawes Scientific Fellowship Award to Kendra Maass to focus on unraveling the mechanistic dependencies of ependymoma tumor cells on extracellular vesicle pathways. Ependymomas are the third most common type of brain tumor in children.
• Discoveries from the 2020 CERN Robert Connor Dawes Pediatric Fellowship were instrumental in helping to win an esteemed $3 million R01 grant from the NCI in 2022 to address significant gaps in our understanding of childhood posterior fossa ependymomas (PFAs).
• In 2021, NBTS’s Research Roundtable hosted a meeting that brought together leading experts in pediatric brain tumor research with FDA, NIH, and biopharma to innovate clinical trials and better utilize patient data.
• In 2021, CERN-funded research and NBTS advocacy culminated in a $6 million U54 grant to develop treatments targeting fusion found in 70% of supratentorial ependymomas. Of the U54 grants made available that year, Drs. Eric Holland and Richard Gilbertson and their team were the only group to receive funding with a focus on pediatric brain tumors.

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

• In 2022, NBTS convened, in collaboration with Liz Salmi and Bethany Kwan, a stakeholder advisory group with over 30 people, including a mix of patients, care partners for pediatric and adult patients, clinicians, and researchers with diverse backgrounds and diagnoses, which has met regularly. Additionally, the advisory group plus additional stakeholders met in May 2023 and will meet once more this year to help in the development of a research agenda focused on quality of life for pediatric patients.
• As part of our Informed in 30 video series, NBTS teamed up with childhood cancer experts Drs. Joanne Porter of Journeys Pediatric Palliative Care and Lauren Weintraub of the Pediatric Neurooncology Program at Albany Medical Center for an educational session that breaks down the concepts, key terms, and questions regarding palliative care in pediatric brain tumors with the help of leading doctors in the field.
• 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 Meditation Mondays, that help families impacted by a pediatric brain tumor diagnosis cope with stress and grief.
• 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 curated 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 helped influence the federal government to:

• Provide $130 million for the Peer-Reviewed Cancer Research Program of the Department of Defense for FY22, a $15 million increase over FY21, and ensured brain cancer, pediatric brain tumors, and pediatric, adolescent, and young adult cancers were once again included as eligible priority research topics for funding.
• Allocate $30 million to fully fund the Childhood Cancer STAR Act for a fourth consecutive year.
• Secure $50 million to fully fund the Childhood Cancer Data Initiative (CCDI) for a third consecutive year.
• Secure $45 billion in funding for the National Institutes of Health (NIH) for FY22, a $2.25 billion increase over FY21, and $6.9 billion to the National Cancer Institute (NCI), a $353 million increase.

In addition, in partnership with our 18,000 advocates across all 50 states, NBTS:

• Held our signature advocacy event, Head to the Hill, to mobilize members of the brain tumor community to share their stories and advocate for the federal government to make strategic investments in brain tumor research and address other critical issues affecting the brain tumor community. One of the issues addressed by participants was urging policymakers to reauthorize the Childhood Cancer STAR Act, which Congress ultimately passed in December 2022. More than 40 advocates from the pediatric brain tumor community participated in the 2023 Head to the Hill.
• Joined the Cancer Moonshot Brain Cancers Forum on glioblastoma (GBM) and diffuse intrinsic pontine glioma (DIPG) at the White House in May 2023. The forum explored ways to accelerate efforts to develop innovative treatments and reduce disparities in access to care and clinical trials for these rare brain cancers.
• Took part in the effort to reignite the Cancer Moonshot, which renewed the Biden administration’s commitment to ensuring progress against cancers, including pediatric brain tumors, remains a national priority.
• Continued support for the Gabriella Miller Kids First Research Act 2.0, which provides funding for a pediatric disease research initiative within the NIH.
• Advocated with other groups for a resolution designating an official DIPG Awareness Day in the United States.

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.