Creating A Standard of Care for Pediatric Brain Tumors – For the First Time

The Defeat Pediatric Brain Tumors Research Collaborative is a powerful, unique global research and drug discovery program which aims to improve clinical outcomes for pediatric brain tumor patients and enable the development of the first-ever standard of care for treating pediatric high-grade gliomas – the most lethal of pediatric cancers. The program infrastructure, researchers, and four interrelated “cores” that make up the program are immediately ready for deployment. All we need is funding.

The program is an NBTS-led research collaborative designed to accelerate research through a platform that fosters collaboration as well as research data, information and materials sharing. The platform consists of four cores that work on critical areas of research simultaneously and in concert with one another to encourage sharing of findings and new discoveries in real time – to aim toward a cure.
Project Impact: A Campaign to Defeat Pediatric Brain Tumors will resource this program by initially providing it with $2.5 million in funding over one year, and $5 million total funding over five years. Funding will support work across four key areas of scientific focus, known as cores:

  • Discovery Core (Molecular Diagnostics & Target Discovery)

    Catalyze the process of discovery to build off of new advances in genetic sequencing and expression

  • Biomarker Core (Identification & Validation):

    Identify and validate biomarkers for pediatric brain tumors across various modalities, including tumor tissue, liquid biopsy (plasma and other body fluids), imaging, etc.

  • Preclinical Modeling & Drug Screening Core:

    Develop the critically needed, relevant mouse models and translate molecular and drug discovery in animal models into targeted therapies that improve patient survival and outcomes

  • Smart Trials Core (N-of-1 Clinical Trials):

    Drive the creation of biology-focused clinical trials, with new findings from the first three cores informing the design, target population, and testing of treatments. Human data from clinical trials will also provide information back to the preclinical cores and seed the development/refinement of new research

Pediatric brain tumors are the leading cause of cancer-related death among children and young adults under age 19. 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, as new discoveries in the lab have led to new treatments in the field that have significantly improved survival.

For example, the most common form of childhood cancer, acute lymphoblastic leukemia, was generally diagnosed as fatal 50 years ago; today, cure rates for the disease reach close to ninety percent.

However, for the 4,600 children diagnosed annually with a brain tumor, the scientific needle has barely moved, with only 25% of children suffering from a malignant brain tumor surviving five years – rates that trend on par with where they were nearly forty years ago.

But, never before in the history of mankind’s battle with cancer has so much progress been made. 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. Among the most transformational of these findings was clear evidence that pediatric high-grade gliomas are biologically and molecularly distinct and separate from their adult counter-parts. 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.

In 2009, NBTS – the largest non-profit funder of brain tumor research in the country – recognized the scientific opportunity to make a difference for patients and families, and committed one-third of its research funding to pediatrics. Since then, NBTS has built a track-record of success, playing a critical role in funding many of the field’s crucial findings that have brought us to the threshold of a new era in 2016. From that strong foundation, we have launched the Defeat Pediatric Brain Tumors Research Collaborative.

The structure of Defeat Pediatric Brain Tumors is what makes it unique.

The research program is based on NBTS’ “Defeat” program model, which is a new business approach to science that facilitates collaborations and exchanges of real-time data to accelerate progress in R&D efforts and transform the way brain tumor research is funded and conducted. This approach is completely unique in its relationship to traditional funding of research by nonprofits, which typically rely on a standard RFA grant-making process, where checks are distributed to individual labs that apply for grant funding, but the research isn’t managed and collaboration isn’t fostered.

The Defeat Pediatric Brain Tumor Research Collaborative approach is beneficial because it:

  • Was not built in a vacuum: the effort is based on input from all key stakeholders –researchers, industry, government, and nonprofits/advocates — to determine the real barriers to getting to more new treatments
  • Is the only effort in this field that is ambitious enough to tackle the challenges via an end-to-end approach, from basic science to the clinic, including powerful international collaborations and integrating with leading clinical trial consortium, the Pacific Pediatric Neuro-Oncology Consortium (PNOC)
  • De-risks early entry of biopharmaceutical companies into pediatric brain tumor trials by producing industry-level data, requisite to beginning new clinical trials in humans
  • Provides 100% of the grant funding to the research: NBTS does not pay for hospital’s indirect costs
  • Doubles the investment dollar: Collaborative agreements signed with partner research centers aim to lock in an agreement that for every dollar of grant investment the institution will match that dollar in cash or in-kind
  • Takes a unique approach to segments like Intellectual Property (IP) to allow breakthroughs to continue to fund the model and expanded research