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Development of drug treatments for neurofibromatosis type 2-associated vestibular schwannoma

Blakeley, Jaishri

Current Opinion in Otolaryngology & Head and Neck Surgery: October 2012 - Volume 20 - Issue 5 - p 372–379
doi: 10.1097/MOO.0b013e328357d2ee

Purpose of review 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.

Recent findings 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.

Summary 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.

Johns Hopkins University, Baltimore, Maryland, USA

Correspondence to Jaishri Blakeley, MD, Assistant Professor of Neurology, Neurosurgery, and Oncology, Johns Hopkins University, 1550 Orleans Street, Suite 1M16, Baltimore, MD 21204, USA. Tel: +1 410 955 8837; fax: +1 410 614 9335; e-mail:

© 2012 Lippincott Williams & Wilkins, Inc.