Available Therapies for Patients with Neurofibromatosis-Related Nervous System Tumors

Curr Treat Options Oncol. 2020 Aug 7;21(10):81. doi: 10.1007/s11864-020-00779-z.

Abstract

Though the majority of nervous system tumors are sporadic, several clinically relevant genetic syndromes are associated with a predisposition to tumors of the central and peripheral nervous system including neurofibromatosis type 1 (NF1), type 2 (NF2), and schwannomatosis (SWN). These represent prototypical tumor suppressor syndromes where loss of a tumor suppressor gene-protein impairs the cell's ability to regulate cell proliferation. While clinical manifestations vary widely for each of these syndromes, tumors arising in the peripheral nerve sheath are a unifying feature. Clinical clues should prompt the clinician to recognize the underlying genetic syndrome and screen for associated tumors including, among others, plexiform neurofibromas and gliomas in NF1 and vestibular schwannomas, meningiomas, and spinal ependymomas in NF2. Improvements in mechanistic understanding of how the genetic mutations that underlie these syndromes contribute to tumor formation have led to new advances in targeted therapies. MEK inhibitors have shown promise for treating progressive plexiform neurofibromas in NF1. Bevacizumab has been shown to improve hearing and treat vestibular schwannomas in NF2. This article reviews the currently available data on management of tumors associated with these three syndromes.

Keywords: Bevacizumab; Neurocutaneous disorders; Neurofibroma; Neurofibromatosis type 1; Neurofibromatosis type 2; Phakomatoses; Schwannomatosis; Vestibular schwannoma.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents, Immunological / therapeutic use*
  • Bevacizumab / therapeutic use*
  • Humans
  • Nervous System Neoplasms / complications
  • Nervous System Neoplasms / drug therapy*
  • Neurilemmoma / complications
  • Neurilemmoma / drug therapy*
  • Neurofibromatoses / complications
  • Neurofibromatoses / drug therapy*
  • Prognosis
  • Skin Neoplasms / complications
  • Skin Neoplasms / drug therapy*

Substances

  • Antineoplastic Agents, Immunological
  • Bevacizumab

Supplementary concepts

  • Schwannomatosis