title: Improving outcomes for neurofibromatosis 1-associated brain tumors.
abstract: Children and adults with neurofibromatosis type 1 (NF1) are predisposed to developing CNS tumors, including optic pathway gliomas (OPGs), brainstem gliomas (BSGs) and high-grade gliomas. Although current first-line treatments for low-grade gliomas (OPGs and BSGs) may prevent further tumor growth, they rarely result in restoration of the associated visual or neurological deficits. The availability of accurate small-animal models of NF1-associated brain tumors has established tractable experimental platforms for the discovery and evaluation of promising therapeutic agents. On the basis of these preclinical studies, biologically targeted agents are now being evaluated in children with NF1-associated low-grade brain tumors. Collectively, these models have also begun to reveal potential neuroprotective and risk assessment strategies for this brain tumor-prone population.
authors: Department of Pediatrics, St. Louis Children's Hospital, St. Louis, MO, USA.
authors: Nicole M
authors: NM
authors: Brossier
authors: David H
authors: DH
authors: Gutmann
mesh_terms: D000328:Adult; D000818:Animals; D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D001933:Brain Stem / Q000473:pathology; D002648:Child; D015195:Drug Design; D005910:Glioma / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D006801:Humans; D058990:Molecular Targeted Therapy; D060787:Neoplasm Grading; D009456:Neurofibromatosis 1 / Q000150:complications*; D020339:Optic Nerve Glioma / Q000188:drug therapy / Q000209:etiology / Q000473:pathology
chemical_list: D000970:Antineoplastic Agents
keywords: RAS; brainstem glioma; cyclic AMP; genetically-engineered mice; mTOR; microglia; neurofibromatosis type 1; optic pathway glioma; preclinical