Ushering in a New Era of Promise for Pediatric Brain Tumor Patients

Why this cause?

Over the past decades, little progress has been made in the treatment of pediatric brain tumors, while tremendous advances have been made across the rest of the pediatric cancer spectrum. For example, the most common form of childhood cancer, acute lymphoblastic leukemia, was generally considered fatal 50 years ago; today, cure rates for the disease reach close to ninety percent. For the more than 4,600 children diagnosed annually with a brain tumor, the scientific needle has barely moved.

  • Five-year survival rates hover between fifteen and thirty percent, yielding survival rates from diagnosis that span only the length of an average school year.
  • Most treatments administered to children with brain tumors were not designed for the developing brain, often leaving survivors with a lifetime of neurological, cognitive, developmental, and other challenges.
  • Lack of preclinical models for effectively testing drugs and small patient population size limit investment by pharmaceutical companies.
  • Global regulatory barriers and a young knowledge base of these tumors inhibit the development of pediatric-specific treatments.

Why now?

The confluence of ground breaking advances in our understanding of pediatric high-grade gliomas and the power of precision medicine leads us to a higher place from which we can launch a concerted fight against pediatric brain tumors. The time to act is now; the opportunity to succeed in improving the lives of children and their families is tremendous.

  • Never before in the history of mankind’s battle with cancer has so much research progress been made.
  • Advances in molecular biology and genetic sequencing have unlocked new knowledge that is informing the development of new treatments and therapies.
  • Through precision medicine, we are working to provide the right therapy for the right person at the right time.
  • Critical findings in recent years – including many funded by the National Brain Tumor Society – have positioned the field of pediatric brain tumors to enter a new era in treatment and survival.
  • These findings allow scientists to break from the norm of the past decades, and catalyze them to develop pediatric-specific treatments that finally move us away from using adult therapies on children, a strategy that simply has not worked.

Why Project Impact?

Project Impact: A Campaign to Defeat Pediatric Brain Tumors, is the National Brain Tumor Society’s push to fund a major translational research and drug discovery program, and create sustainable support for pediatric brain tumor research and clinical trials. Project Impact, as a capital campaign, seeks to initially raise at least $2.5 million in speciality gifts and funds over five years to resource the launch of scientific projects within the Defeat Pediatric Brain Tumors Research Collaborative, with an ongoing aim to ultimately invest more than $5 million, from multiple sources, in the program. Through this paradigm-shifting strategy, NBTS will fundamentally – and permanently – change the trajectory of pediatric high-grade gliomas by developing:

  • A new standard of care that deploys effective drugs to dramatically extend the survival of children with high-grade gliomas.
  • A new methodology of conducting research differently – breaking the old mold of single-investigator-driven projects and moving to highly collaborative team-science where sharing data in real time is fueling faster research results.
  • New and trusted preclinical models for testing that incentivize the pharmaceutical industry to provide compounds and agents to researchers
  • A research platform that creates a system of tested and validated compounds to feed Phase I clinical trials.
  • A better understanding of how epigenetic changes drive tumor development, progression, and resistance.
  • A collaborative research model that is scalable across the pediatric cancer spectrum that discovers new knowledge that can be applied in the fight against all pediatric brain tumors.