title: Development of drug treatments for neurofibromatosis type 2-associated vestibular schwannoma.
abstract: OBJECTIVE
  To review the discoveries in molecular pathophysiology contributing to the development of neurofibromatosis type 2 (NF2)-associated vestibular schwannomas and the recent experiences with drug therapies for these tumors. The review includes discussion of diagnostic criteria for NF2, populations to clinically consider for drug therapy and drug targets currently under consideration for NF2.
  
  
  RESULTS
  Increased insight into the complex pathways that underlie both the genetic syndrome of NF2 and the specific pathogenesis of vestibular schwannomas has highlighted multiple potential therapeutic targets. These discoveries have been translated into clinical trials with some early promising results. Inhibition of angiogenesis as well as regulation of mammalian target of rapamycin and the epidermal growth factor receptor family of receptors are the focus of current clinical investigations.
  
  
  CONCLUSIONS
  Although a great deal of work is ongoing to understand the multiple effects of the lack of the regulating protein Merlin on tumorgenesis in patients with NF2, advances are ongoing with clinical therapeutics. There is cause for enthusiasm based on recent results with antiangiogenesis therapy in select patients with NF2 and progressive vestibular schwannomas; however, awareness of the notable risks and limitations of therapies currently in development is required.
authors.affiliation: Johns Hopkins University, Baltimore, Maryland, USA. jblakel3@jhmi.edu
authors.forename: Jaishri
authors.initials: J
authors.lastname: Blakeley
mesh_terms: D020533:Angiogenesis Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D005260:Female; D005500:Follow-Up Studies; D005544:Forecasting; D006801:Humans; D008297:Male; D058990:Molecular Targeted Therapy / Q000379:methods*; D009389:Neovascularization, Pathologic / Q000517:prevention & control*; D016518:Neurofibromatosis 2 / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D025581:Neurofibromin 2 / Q000187:drug effects / Q000235:genetics; D009464:Neuroma, Acoustic / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D018570:Risk Assessment; D058570:TOR Serine-Threonine Kinases / Q000187:drug effects / Q000235:genetics; D016896:Treatment Outcome
chemical_list: D020533:Angiogenesis Inhibitors; D025581:Neurofibromin 2; D058570:TOR Serine-Threonine Kinases