Targeted therapies for the neurofibromatoses

Lauren D. Sanchez, Ashley Bui, Laura J. Klesse

Research output: Contribution to journalReview articlepeer-review

12 Scopus citations


Over the past several years, management of the tumors associated with the neurofibromatoses has been recognized to often require approaches that are distinct from their spontaneous counterparts. Focus has shifted to therapy aimed at minimizing symptoms given the risks of persistent, multiple tumors and new tumor growth. In this review, we will highlight the translation of preclinical data to therapeutic trials for patients with neurofibromatosis, particularly neurofibromatosis type 1 and neurofibromatosis type 2. Successful inhibition of MEK for patients with neurofibromatosis type 1 and progressive optic pathway gliomas or plexiform neurofibromas has been a significant advancement in patient care. Similar success for the malignant NF1 tumors, such as high-grade gliomas and malignant peripheral nerve sheath tumors, has not yet been achieved; nor has significant progress been made for patients with either neurofibromatosis type 2 or schwannomatosis, although efforts are ongoing.

Original languageEnglish (US)
Article number6032
Issue number23
StatePublished - Dec 1 2021


  • Low grade glioma
  • Neurofibromatosis
  • Plexiform neurofibroma
  • Vestibular schwannoma

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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