Targeted therapy on neurofibromatosis type 2: Current state of clinical trials

Abstract

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 cer-tain signs and symptoms of NF2, such as hearing loss. We review the current state of clinical trials of NF2.