Children’s National Hospital Receives $8 Million Grant from Gilbert Family Foundation to Advance Neurofibromatosis Research
Curing NFPress ReleaseOct 09, 2025
The investment deepens a partnership to transform care for one of the most common genetic conditions in children
WASHINGTON (Oct. 9, 2025) – 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:
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.
Media contact: media@childrensnational.org | 202-476-4500
###
About Children’s National Hospital
Children’s National Hospital, based in Washington, D.C., was established in 1870 to help every child grow up stronger. Today, it is one of the top 10 children’s hospitals in the nation and ranked in all specialties evaluated by U.S. News & World Report. Children’s National is transforming pediatric medicine for all children. The Children’s National Research & Innovation Campus opened in 2021, a first-of-its-kind pediatric hub dedicated to developing new and better ways to care for kids. Children’s National has been designated four times in a row as a Magnet® hospital, demonstrating the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty care locations in the D.C. metropolitan area, including Maryland and Virginia. Children’s National is home to the Children’s National Research Institute and Sheikh Zayed Institute for Pediatric Surgical Innovation. It is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. In 1987, Children’s National founded Safe Kids Worldwide, a non-profit dedicated to reducing unintentional injuries among children through comprehensive national and global education, research and advocacy. As a non-profit, Children’s National relies on generous donors to help ensure that every child receives the care they need.
For more information, follow us on Facebook, Instagram and LinkedIn.
Innovation District Post
Advancing innovative treatments for neurofibromatosis type 1
Date TBD
A new $8 million investment from the Gilbert Family Foundation will support groundbreaking research on neurofibromatosis type 1 (NF1) at Children’s National Hospital. The generous five-year grant continues the Foundation’s longstanding research partnership with the hospital. It will accelerate investigations aimed at improving the lives of children and young adults affected by this genetic condition.
“This grant will enable us to expand basic science, translational and clinical research. It will help us perform two to three innovative clinical trials in the next five years — trials we will share with the NF community as a whole,” says Roger J. Packer, MD, the Gilbert Family Distinguished Professor of Neurofibromatosis at Children’s National.
Dr. Packer will lead the research team, which also includes Javad Nazarian, PhD, Steven Stasheff, MD, Karin S. Walsh, PsyD and Sridevi Yadavilli, MD, PhD.
Why this matters
NF1 is one of the most common genetic conditions in young people. It affects approximately 1 in 3,000 children born 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.
Dive deeper
The new funding will:
Children’s National Leads the Way
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.
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.
Gilbert Family Neurofibromatosis Institute accomplishments include: