Washington, DC — October 9, 2025 — Leads & Copy — Children’s National Hospital and the Gilbert Family Foundation have announced an $8 million grant to support research on neurofibromatosis type 1 (NF1). The five-year grant from Gilbert Family Foundation’s Brain Tumor Initiative (BTI) will accelerate investigations aimed at improving the lives of children and young adults affected by this genetic condition, which affects approximately 1 in 2,500 births each year, causing nervous system tumors and lifelong health complications, including vision and learning disabilities, skeletal abnormalities and increased cancer risks.
The grant will support projects to address these challenges, including clinical trials testing new therapies that combine MEK inhibitors, which block tumor growth, and checkpoint inhibitors, which help the immune system attack cancer, to treat aggressive NF1 brain tumors. It will also drive cognitive research to understand learning, behavior and social deficits in children with NF1; better detect and understand vision loss in children with optic pathway tumors; and build novel lab models to supercharge drug discovery, including the development of NF1 zebrafish models and patient-derived organoids.
In 2007, Dan and Jennifer Gilbert established the Gilbert Family Neurofibromatosis Institute at Children’s National, and since its founding in 2015, the Gilbert Family Foundation has continued to partner with Children’s National to further the fight against NF1.
Roger J. Packer, MD, the Gilbert Family Distinguished Professor of Neurofibromatosis at Children’s National, said the partnership enables them to bring new ideas to the field and apply them safely, helping more patients with NF1 lead successful, independent and happy lives.
Kalyan Vinnakota, PhD, director of Curing NF at Gilbert Family Foundation, said the Children’s National team has made progress in treating NF1-associated gliomas, and they look forward to further innovation and life-changing research. Poornima Venkat, PhD, scientific program manager of Gilbert Family Foundation’s BTI, added that through new research, lab models and clinical trials, they are developing more targeted treatments for NF1 and accelerating toward a cure.
Media Children’s National Hospital 2024764500 Media@childrensnational.org
Source: Children’s National Hospital
