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

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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.

Summary

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.

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Authors and Affiliations

  1. Department of Otolaryngology-Head & Neck Surgery, Indiana University School of Medicine, Fesler Hall Suite 400 1130 W. Michigan Street, Indianapolis, IN, 46202, USA

    Evan C. Cumpston & Charles W. Yates

  2. Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA

    Steven D. Rhodes

  3. Department of Neurosurgery, Indiana University School of Medicine, Indianapolis, IN, USA

    Charles W. Yates

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  1. Evan C. Cumpston
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  2. Steven D. Rhodes
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  3. Charles W. Yates
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Correspondence to Charles W. Yates.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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Evan Cumpston and Steven Rhodes declare no conflicts of interest. Charles Yates has served on the Board of Directors for St. Joseph Institute for the Deaf and has served as an expert witness for Aylstock, Witkin, Kreis & Overholtz PLLC.

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Cumpston, E.C., Rhodes, S.D. & Yates, C.W. Advances in Targeted Therapy for Neurofibromatosis Type 2 (NF2)-Associated Vestibular Schwannomas. Curr Oncol Rep 25, 531–537 (2023). https://doi.org/10.1007/s11912-023-01388-3

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