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 is 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.

The harsh realities of pediatric brain tumors are set against the promising backdrop of advances and innovation in the research of pediatric cancers as a whole. Through the power of molecular biology and genetic sequencing, we now know more than ever before about the makeup and biology of these tumors.

And by applying that knowledge, new therapies could be developed that not only improve a child’s overall survival, but could lead to new treatments that can offer cures.

The research program is based on NBTS’ “Defeat” program model, which is a new business approach to science that facilitates collaboration 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.
• 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, not to any of the research institutions 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.
• A unique approach to segments like Intellectual Property (IP) to allow breakthroughs to continue to fund the model and expanded research.