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Review
. 2023 May;25(5):531-537.
doi: 10.1007/s11912-023-01388-3. Epub 2023 Mar 18.

Advances in Targeted Therapy for Neurofibromatosis Type 2 (NF2)-Associated Vestibular Schwannomas

Evan C Cumpston  1 , Steven D Rhodes  2 , Charles W Yates  3   4
Affiliations
Review

Advances in Targeted Therapy for Neurofibromatosis Type 2 (NF2)-Associated Vestibular Schwannomas

Evan C Cumpston et al. Curr Oncol Rep. 2023 May.

Abstract

Purpose of review: Neurofibromatosis 2 (NF2) is an autosomal-dominant genetic disorder characterized by bilateral vestibular schwannomas (VS), meningiomas, ependymomas, spinal and peripheral schwannomas, optic gliomas, and juvenile cataracts. Ongoing studies provide new insight into the role of the NF2 gene and merlin in VS tumorigenesis.

Recent findings: As NF2 tumor biology becomes increasingly understood, therapeutics targeting specific molecular pathways have been developed and evaluated in preclinical and clinical studies. NF2-associated VS are a source of significant morbidity with current treatments including surgery, radiation, and observation. Currently, there are no FDA-approved medical therapies for VS, and the development of selective therapeutics is a high priority. This manuscript reviews NF2 tumor biology and current therapeutics undergoing investigation for treatment of patients with VS.

Keywords: Medical therapy; Molecular targeted drug therapy; Neurofibromatosis type 2; Selective therapeutics; Vestibular schwannoma.

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