Advancing innovative treatments for neurofibromatosis type 1
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 2,500 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:
- 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.
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:
- Leading one of the world’s foremost NF research networks: the Department of Defense-funded Neurofibromatosis Clinical Trials Consortium
- Becoming one of the leading programs in the Sarcoma Alliance for Research through Collaboration (SARC Alliance) for developing new molecularly-targeted therapies
- Being recognized by the Children’s Tumor Foundation as a Neurofibromatosis Center of Excellence
