The Children’s Hospital of Philadelphia (CHOP) has secured funding of $10m from the Gilbert Family Foundation to help accelerate neurofibromatosis research.
Dan and Jennifer Gilbert established the Gilbert Family Foundation to accelerate a cure for neurofibromatosis type 1 (NF1).
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The hospital will use the funding to support its neurofibromatosis programme, which includes studies to develop guidelines for the management of children with NF1 brain tumours occurring outside of the visual pathway.
The multi-institutional project, led by CHOP, will focus on capturing data that will help create these guidelines and inform further epidemiological studies.
In addition, research will include studies to determine clinical and molecular risk factors for the development and outcomes of NF1-related malignant brain tumours including high-grade glioma and high-grade astrocytoma with piloid.
As part of the project, researchers are planning to use computational methods and new approaches to identify a unique tumour antigen that can be targeted using CAR T-cell therapy.
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By GlobalDataCHOP Neurofibromatosis Program director and Neuro-Oncology Section chief Michael Fisher said: “Research is an essential component of our Neurofibromatosis Programme. Our multidisciplinary team of experts in neuro-oncology, neuro-ophthalmology, neurology, genetics, paediatrics, ophthalmology, orthopaedics and other specialities seek to improve the care and outcomes of those with neurofibromatosis.
“These grants from the Gilbert Family Foundation will allow us to conduct crucial studies that we hope will eventually lead to better treatments for those with NF1,” Fisher added.
The Chop-led project will also focus on understanding the prevalence of malignant transformation of NF1-associated low-grade glioma in adults and evaluate prevalent treatment methods.
Gilbert Family Foundation executive director Laura Grannemann said: “We are proud of our continued investment in CHOP, one of the premier research institutions in the world and a facility that continues to be on the cutting edge of NF research.
“Through our ongoing collaboration with CHOP’s Neurofibromatosis Program, we are confident that we are closer than ever to finding a cure for NF.”
