Investigate and Target an Inflammatory Response During NF1-OPG Formation
Abstract
Pediatric low-grade glioma (pLGG) is the most common brain tumor in children, but rarely occurs in adults. About 14 percent of pLGGs arise from individuals with neurofibromatosis type 1 (NF1), which frequently develop along the optic pathway and thus are also known as NF1-associated optic pathway gliomas (NF1-OPGs). As the most common tumor in the central nervous system (CNS), OPGs develop in nearly 20 percent of children with NF1 and are predominately diagnosed in patients younger than 7 years of age with few, if any, adult cases. Thus, both sporadic and NF1-associated pLGGs appear to arise from the transient vulnerability to hyperactive RAS-mediated ERK/MAPK signaling in neural stem and progenitor cells in the developing CNS.While most NF1-OPG patients have excellent survival, approximately 30%, despite conventional chemotherapies, will experience vision loss, highlighting an unmet clinical need for novel therapies. Recent imaging studies have shown that loss of retinal ganglion cells (RGCs) the only neuronal population connecting the eye to the brain is associated with vision loss inNF1-OPG patients.
Document Details
- Document Type
- Technical Report
- Publication Date
- Jul 01, 2023
- Accession Number
- AD1227037
Entities
People
- Fransico Nadal-nicolas
- Kiyoharu J. Miyagishima
- Wei Li
- Wenxin Ma
Organizations
- National Eye Institute