National Brain Tumor Society Names Leaders for Pediatric-Focused Research Effort

Top minds in field of pediatric neuro-oncology to advise new research collaborative

National Brain Tumor Society (NBTS), the largest nonprofit organization dedicated to the brain tumor community in the United States, today announced a new committee of distinguished pediatric oncologists who will advise the organization’s Defeat Pediatric Brain Tumors Research Collaborative. The leaders will form the Strategic Scientific Advisory Committee (SSAC) for Defeat Pediatric Brain Tumors, and will be charged with working with NBTS staff to oversee the direction of the research and guiding its four cores to ensure milestones are being met and that the most promising discoveries are advancing toward the clinic.

Scientific Director Roger Packer, MD of Children’s National Medical Center, will lead the SSAC, which also includes:

• Susan Blaney, MD – Deputy Director of the Texas Children’s Cancer and Hematology Centers; Executive Vice Chair, Department of Pediatrics, Baylor College of Medicine (BCM); Vice President, Clinical and Translational Research, BCM; and holds the Martha Ann and Harold M. Selzman, M.D. Endowed Chair in the Institute for Clinical and Translational Research at BCM; Vice Chair of the Children’s Oncology Group.
• Richard Gilbertson, MD, PhD – Li Ka Shing Chair of Oncology and Director of the Cambridge Cancer Centre (England)
• Scott Pomeroy, MD, PhD – Neurologist-in-Chief and Chairman, Department of Neurology, Boston Children’s Hospital; Consultant, Pediatric Neuro-Oncology, Dana-Farber Cancer Institute; Bronson Crothers Professor of Neurology, Harvard Medical School
• Raphaël Rousseau, MD, PhD – Group Medical Director / Global Franchise Head, Pediatrics, Genentech, a member of the Roche Group
• Robert Wechsler-Reya, PhD – Director, Tumor Initiation and Maintenance Program, Sanford-Burnham Medical Research Institute in La Jolla, CA

“Although the past several years have marked a period of incredible advancement in our understanding of the biology of pediatric high-grade gliomas, this body of knowledge has not yet translated into better treatment options for this vulnerable patient population,” said Carrie Treadwell, National Brain Tumor Society’s Chief Research Officer and the President of Pediatric Cancer Cure, LLC, an NBTS subsidiary that administers the organization’s latest pediatric initiative. “This group of advisors, by way of their expertise and guidance, will be a major asset to the National Brain Tumor Society’s efforts to change the narrative for these difficult to treat pediatric brain tumors.”

The Defeat Pediatric Brain Tumors Research Collaborative is the research arm of the National Brain Tumor Society’s current pediatric brain tumor initiative, Project Impact: The Campaign to Defeat Pediatric Brain Tumors. Project Impact is a precision medicine model for pediatric cancer drug development that combines efforts in funded research and public policy to confront inefficiencies that exist in both the pediatric brain tumor preclinical research system and pediatric cancer clinical trial environment. These two tracks will work together to create a preclinical research platform and regulatory strategy to de-risk early entry of biopharmaceutical companies into pediatric brain tumor trials and inform standard of care. Together, these efforts will seek to optimize the entire pediatric brain cancer drug discovery and development pipeline and create significant advances in treatment options for children with brain tumors.

Defeat Pediatric Pediatric Brain Tumors consists of four cores, which will focus on discovery science, biomarker identification, creation of informative models, and expedited therapeutic intervention through both preclinical testing and smart clinical trials. This highly integrated tactic has the goal to move science into the clinic in the next five years through the power of collaboration. NBTS believes this approach can serve as a model for future expansion into other pediatric brain tumor types.

This year in the U.S., an estimated 4,620 infants, children, and adolescents will receive a brain tumor diagnosis. This rate of occurrence, unfortunately, now places brain tumors atop the list most common cancers in the pediatric population. Of these cases, approximately 20% will be given the diagnosis of an aggressive class of brain tumors known as pediatric high-grade gliomas (pHGGs). Tragically, pHGG patients are faced with five-year survival rates of only 15-30%. Even more devastating, a unique subgroup of pHGG patients diagnosed with a tumor known as diffuse intrinsic pontine glioma (DIPG) have a mean survival of only nine months. These rates have remained relatively unmoved in more than 40 years, yet there has still never been a drug developed specifically to treat pediatric brain tumors.