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Targeted therapy on neurofibromatosis type 2: Current state of clinical trials
J Korean Skull Base Soc -0001;():-
Published online December 21, 2022
© 2022 Korean Skull Base Society.

Gyeong Hwa Jeon1, Jong Dae Lee2

1Department of Otorhinolaryngology-Head and Neck Surgery, Soonchunhyang University Seoul Hospital, Seoul, Korea
2Department of Otorhinolaryngology-Head and Neck Surgery, Soonchunhyang University Bucheon Hospital, Bucheon, Korea
Correspondence to: Jong Dae Lee
Received August 17, 2022; Revised September 13, 2022; Accepted September 16, 2022.
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License ( which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Neurofibromatosis type 2 (NF2) is a rare autosomal dominant disorder characterized as bilateral vestibular schwannoma (VS), various brain and spinal tumors. Therapeutic options for NF2 patients have been limited to surgery and radiation. However, outcomes are not effective for NF2-related VSs. Understanding the molecular mechanisms driving NF2 pathogenesis holds promise for the potential use of targeted therapy. Many targeted therapies have been evaluated in preclinical models. Several clinical trials have been conducted to stop tumor growth and, in some cases, to improve certain signs and symptoms of NF2, such as hearing loss. We review the current state of clinical trials of NF2.
Keywords : Vestibular schwannoma, Molecular targeted therapy, Clinical trial

September 2022, 17 (2)
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