Funds will support research to identify promising treatments for patients with NF1
PHILADELPHIA, March 28, 2023 – Children’s Hospital of Philadelphia (CHOP) today announced that it has received more than $10 million in a series of gifts to fund critical neurofibromatosis research from the Gilbert Family Foundation, a private foundation established by Dan and Jennifer Gilbert to accelerate a cure for neurofibromatosis type 1 (NF1).
Neurofibromatosis type 1 is a genetic disorder that can affect multiple systems of the body and is characterized by symptoms that include skin changes, skeletal abnormalities and tumors, both benign and malignant. NF1 occurs in approximately one in 3,000 people and affects males and females of all races equally. Because neurofibromatosis type 1 is hereditary, the genes causing the condition may be passed from generation to generation in a family. However, the type and severity of this disorder can vary widely among affected family members.
The funds will support neurofibromatosis research via CHOP’s Neurofibromatosis Program in a variety of ways, including:
· Studies for the Brain Tumor Initiative, launched by the Gilbert Family Foundation, which will focus on identifying and developing therapeutic strategies to treat NF1-associated transformed gliomas. Gliomas are tumors that begin in the brain or spinal cord and are the most common primary central nervous system (CNS) tumors in NF1 patients. The objective is to increase the understanding of the biology and behavior of gliomas in patients with NF1, ultimately improving the care of patients with NF1 who develop these tumors.
· Studies to better characterize and understand the deterioration of the visual system that occurs in NF1 patients who develop optic pathway gliomas (OPGs). This includes identifying the most meaningful and robust visual biomarkers needed to evaluate future neuroprotection and visual restoration treatments. The long-term goal is to improve and restore vision in children with NF1 who develop optic pathway gliomas (OPGs), a low-grade glioma that involves one or more structures of the anterior visual pathway. OPGs can cause permanent vision loss ranging from a mild decline to complete blindness. The cause of OPG vision loss is unknown, so establishing these metrics and biomarkers will be critical to evaluate vision restoration strategies.
· Research that will lead to the discovery of immunotherapy targets for NF1-associated high-grade glioma (HGG), a malignant brain tumor that sometimes develops in NF1 patients and currently lacks effective therapies. Using computational methods and novel approaches, the researchers hope to identify a unique tumor antigen that can be targeted using CAR T-cell therapy.
“Research is an essential component of our Neurofibromatosis Program. Our multidisciplinary team of experts in neuro-oncology, neuro-ophthalmology, neurology, genetics, pediatrics, ophthalmology, orthopedics and other specialties seek to improve the care and outcomes of those with neurofibromatosis” said Dr. Michael Fisher, Section Chief of Neuro-Oncology and Director of the Neurofibromatosis Program at CHOP. “These grants from the Gilbert Family Foundation will allow us to conduct crucial studies that will eventually lead to better treatments for those with NF1.”
The Gilbert Family Foundation was founded in 2015 to help accelerate a cure for NF. Nick Gilbert – the oldest son of Dan and Jennifer Gilbert – was diagnosed with NF1 as an infant and continues to battle the disease. The organization launched its Brain Tumor Initiative (BTI) in 2022. This initiative joins the Vision Restoration and Gene Therapy initiatives, which are focused on addressing different NF manifestations and finding an underlying cure for the disease. BTI focuses on identifying and developing therapeutic strategies to effectively treat NF1-associated transformed gliomas. BTI seeks to conduct studies to provide critical characterization of NF1 transformed gliomas and identify the most promising treatments for patients.
“We are proud of our continued investment into CHOP, one of the premier research institutions in the world and a facility that continues to be on the cutting edge of neurofibromatosis research,” said Laura Grannemann, the Executive Director of the Gilbert Family Foundation. “Through our ongoing partnership with CHOP, we are confident that we are closer than ever to finding a cure for NF.”
CHOP was in the first cohort of research partners to receive funding for BTI and has also received previous investments for its promising research as part of the Gilbert Family Foundation’s Vision Restoration Initiative (VRI).
To find out more information on the work in the space of visit chop.edu/centers-programs/neurofibromatosis-program and research.chop.edu.
About Children’s Hospital of Philadelphia
A non-profit, charitable organization, Children’s Hospital of Philadelphia was founded in 1855 as the nation’s first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the 595-bed hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as a new inpatient hospital with a dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children’s Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu.
About the Gilbert Family Foundation
The Gilbert Family Foundation is a private nonprofit foundation founded by Jennifer and Dan Gilbert to accelerate a cure for neurofibromatosis type 1 (NF1) and build economic opportunity and equity in the city of Detroit. The Gilbert Family Foundation supports groundbreaking, cutting-edge research in hopes of eradicating NF1, a genetic disorder that causes tumors to grow on nerves anywhere in the body. Additionally, the Foundation supports the economic stability and mobility of residents in Detroit by investing in wealth-building opportunities and breaking down systemic barriers. For more information on the Gilbert Family Foundation, please visit gilbertfamilyfoundation.org.