Philanthropy, Collaboration, and Advocacy Overcome Roadblocks at the Intersection of Cancer and Rare Disease

Hearing the words “You have cancer” is life-changing—even more so when your cancer is one that doctors very rarely see. This is especially true for children who have brain tumors because childhood brain cancer is already rare, and no two tumors are exactly alike. As with any rare disease, childhood brain cancer has historically presented challenges to research and treatment: Scientists and physicians have few cases on which to base their understanding of the disease, and research and treatment development are often slowed by insufficient funding. As a result, patients often face challenging diagnoses and suboptimal treatment options.

However, having worked within the pediatric and rare oncology space for many years, I see pivotal opportunities for advances in rare cancer research and treatment through philanthropic funding, patient advocacy, and cross-sector collaboration. Advocates and foundations are playing a central role in improving the therapeutic development process and introducing more treatments by driving collaboration across sectors. I lead the Rare Disease and Oncology portfolio within the Science Philanthropy Accelerator for Research and Collaboration (SPARC) program, part of the Milken Institute’s Strategic Philanthropy pillar, and through my work, I have experienced the critical bridges that are being developed by patient families and advocates to drive this tide change:

• Collaboration among patient advocates, nonprofits, and pharma was critical for Food and Drug Administration (FDA) approval of a targeted therapy for children with low-grade glioma. For many medical conditions, therapies are approved for adult patients for years, or even decades, before they are approved or even researched for children. It was thus a triumph when, in 2024, the FDA approved a drug for children with a subset of brain cancer called low-grade glioma (LGG). The drug’s journey in pediatric low-grade glioma (pLGG) started with a group of families who raised money specifically to address the limited treatment options for children with LGG. Starting with the significant and sustained funding that supported academic research, the foundation formed by these families in 2005 collaborated with nonprofits, pharmaceutical companies, academics, and others to support and advocate for research that led to clinical trials of tovorafenib in children.
  
  In 2023, I co-chaired a workshop alongside leaders from nonprofit organizations, academia, and the pharmaceutical industry, including the patient advocacy leadership from the company that advanced the drug’s development. The workshop examined the people, processes, and partnerships that advanced tovorafenib development in pLGG. The resulting report revealed that nonprofit organizations play a central role in promoting collaboration among stakeholders, and patients and their families impact every stage of pediatric cancer research—from funding academic research labs to providing firsthand insights about the patient experience and helping investigators gain support in patient communities—all of which are essential for successful drug development.
   
• A philanthropic investment can incentivize collaboration and data sharing to enable discoveries in rare brain tumors. In 2022, Children’s National Hospital launched the International Rare Brain Tumor Registry, a collaboration among 14 hospitals worldwide. This initiative, made possible by a large philanthropic investment, collects biological samples and data from children with the rarest brain tumors to identify their causes and potential drug targets. With fewer than 10 cases of many tumor types at its inception, the registry has since amassed data from more than 200 cases across ultra-rare tumor types, providing researchers with the large datasets that they need to better understand how to fight these cancers. SPARC was proud to partner with Children’s National and the donor, identifying the unique opportunities for philanthropic capital to drive progress in childhood brain cancer.
   
• Sustained funding enables the rapid launch of clinical trials, offering hope to children who have exhausted standard therapy options. The Pediatric Neuro-Oncology Consortium (PNOC), formed in 2012, brings together over 275 clinicians and researchers to accelerate breakthroughs in childhood brain cancer treatment. Members share data as they develop efficient clinical trials that test highly targeted therapies. To date, PNOC has enrolled over 900 patients in dozens of clinical trials. The consortium’s efficiency is made possible by its largely philanthropic support, which provides the sustained financial independence to conduct clinical trials quickly in a way that traditional funding mechanisms cannot.
  
  PNOC and the PNOC Foundation represent a highly collaborative and innovative relationship that enables the conduct of critical brain cancer clinical trials. The Day One Biopharmaceuticals clinical trials, as well as several from the Brain Tumor Institute at Children’s National, are run through PNOC, emphasizing the important role of this model in advancing childhood brain cancer clinical trials.

This exciting work in rare cancer highlights common themes behind recent progress, which the SPARC Rare Disease and Oncology program leverages to improve philanthropic giving strategies. Collaboration is the most significant impact multiplier, and it should be expected from not only academic researchers but also all individuals, companies, and institutions committed to changing the status quo for rare diseases.