When you have a rare disease, you face two battles. The illness itself, and the reality of living in a world where no one knows what you are up against.

Brain cancer accounts for a quarter of total cancer diagnoses and is the leading cause of cancer death in children and teens. Complicating brain cancer research even more is that it is further subdivided into over twelve different types and over one hundred subtypes, which has so far put significant progress toward better treatments out of reach.

Approximately 5,000 children and teens in the U.S. are diagnosed each year. Due to a limited scientific understanding of rare cancer subtypes, patients who are diagnosed with these cancers often have worse outcomes. 

A new grant of nearly $100,000,000 in philanthropic funding from an anonymous donor to Children’s National Hospital in Washington, DC was announced in June 2023. Dedicated to the improvement of research and clinical care for children with brain cancer, this investment is a welcome infusion of capital for childhood cancers that devastate children, their families, and entire communities.

This single investment nearly matches grant funding for pediatric brain cancer research granted by the National Cancer Institute in a year, bringing capital into a field that receives a fraction of the 4% to 8% of federal funding dedicated to childhood cancer research. 

What makes this contribution unique is not only its size, but how the grant program has been designed. It is setting out to address all aspects of health and wellbeing for children diagnosed at Children’s National Hospital and far beyond. It will provide capital to conduct research to develop safer and more effective therapies for children with brain cancer. It will also fund the development of programs that nurture patient and family resilience and well-being. As philanthropists consider how to maximize the benefits of their giving, there are a few lessons this investment can offer as it pertains to structure and governance. Successful investments in research should:

  • Engage external advisors to ensure scientific rigor and accountability;
  • Support a diverse range of investigators committed to open science in order to ensure diversity of thought within and external to project teams; and, 
  • Fund research that provides critical data and resources the research community can utilize beyond the term of the grant to ensure the development and solidification of research ideas that can compete for federal funding. 

Coupled with a program designed to focus on building infrastructure for international collaboration and information-sharing, this transformative program is poised to create a fundamental shift in the understanding of the rarest childhood brain tumors. Most donors come to biomedical philanthropy when confronted first-hand with the reality of a devastating diagnosis. As moonshot philanthropy gains traction in response to the desire to create meaningful impact in health, it will be important to carefully consider not only the desired outcome of the funding but also the design of the program to ensure that outcome is achieved.