Children’s National Hospital Receives $8 Million Grant From Gilbert Family Foundation to Advance Neurofibromatosis Research

Children's National Hospital and the Gilbert Family Foundation today announced a new $8 million, five-year grant to support groundbreaking research on neurofibromatosis type 1 (NF1). The grant, which is part of 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.

NF1 affects approximately 1 in 2,500 births each year. It causes potentially deadly nervous system tumors and can pose lifelong health complications. These include vision and learning disabilities, skeletal abnormalities and increased cancer risks. The grant will support a series of ambitious projects to address these challenges.

"The Gilbert Family Foundation is leading a global movement to transform how we care for NF1. Their partnership enables us to bring new ideas to the field and apply them safely. This will help more patients with NF1 lead successful, independent and happy lives," said Roger J. Packer, MD, the Gilbert Family Distinguished Professor of Neurofibromatosis at Children's National.

The new funding will:

• Fuel 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. Trials will target high-grade astrocytomas with piloid features (HGAPs) and recalcitrant gliomas. The goal is to improve outcomes for these aggressive tumors by enhancing the body's immune response to cancer.
• 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. Discoveries will help doctors predict, treat and prevent disease progression as well as assess the potential benefits of treatment.
• Build novel lab models to supercharge drug discovery. The grant will support the expansion and validation of investigational models, including the development of NF1 zebrafish models and patient-derived organoids. NF1 tumor organoid tissues, grown in the lab, mimic the functioning of an actual organ.

In 2007, Dan and Jennifer Gilbert established the Gilbert Family Neurofibromatosis Institute at Children's National. The Institute advances basic and clinical research, providing a global destination for care. The Gilbert family endowed three professorships that help the hospital advance the field. Since its founding in 2015, the Gilbert Family Foundation has continued to partner with Children's National to further the fight against NF1.

"The Children's National team has made significant progress in treating NF1-associated gliomas as a part of Gilbert Family Foundation's BTI," said Kalyan Vinnakota, PhD, director of Curing NF at Gilbert Family Foundation. "We look forward to further innovation and life-changing research through the expansion of this partnership."

"This new award is a significant addition to the BTI portfolio," added Poornima Venkat, PhD, scientific program manager of Gilbert Family Foundation's BTI. "Through new research, lab models and clinical trials, we are developing more targeted treatments for NF1 and accelerating toward a cure."

Previous funding from the Gilbert Family Foundation has improved our understanding of NF1-related gliomas and how best to treat them. It has supported pioneering work in evaluating the safety and cognitive benefits of novel treatments, primarily MEK inhibitors. The Foundation's new award will build upon this work. It will yield new insights into one of the most lethal manifestations of NF1: transformation of low-grade gliomas into malignant lesions. Children's National will explore novel therapies, including immunotherapy, with the potential to save lives.