Neurofibromatosis type 1 and optic pathway glioma: Molecular interplay and therapeutic insights

Abstract

Children with neurofibromatosis type 1 (NF1) are predisposed to develop central nervous system neoplasms, the most common of which are low-grade gliomas (LGGs). The absence of human NF1 associated LGG-derived cell lines, coupled with an inability to generate patient-derived xenograft models, represents barriers to profile molecularly targeted therapies for these tumors. Thus, genetically engineered mouse models have been identified to evaluate the interplay between Nf1-deficient tumor cells and nonneoplastic stromal cells to evaluate potential therapies for these neoplasms. Future treatments might also consider targeting the nonneoplastic cells in NF1-LGGs to reduce tumor growth and neurologic morbidity in affected children.

Keywords: genetically engineered mouse models; neoplastic cells; neurofibromatosis 1; nonneoplastic stromal cells; optic pathway glioma; therapeutic insights.