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Review
. 2021 Nov 30;13(23):6032.
doi: 10.3390/cancers13236032.

Targeted Therapies for the Neurofibromatoses

Lauren D Sanchez  1 , Ashley Bui  2 , Laura J Klesse  2
Affiliations
Review

Targeted Therapies for the Neurofibromatoses

Lauren D Sanchez et al. Cancers (Basel). .

Abstract

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.

Keywords: low grade glioma; neurofibromatosis; plexiform neurofibroma; vestibular schwannoma.

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Conflict of interest statement

L.J.K. has served on a medical advisory board for AstraZeneca.

Figures

Figure 1
Figure 1
Schematic representation of the main NF1 associated signaling pathways with noted targeted therapies. RTK = receptor tyrosine kinase. Figure created with biorender.com (accessed on 19 October 2021).
Figure 2
Figure 2
Simplified schema of the signaling pathways implicated in tumor formation with loss of merlin, the protein product of NF2. Therapies utilized to target these activated pathways are noted. This figure was created with Biorender.com (accessed on 19 October 2021).
See this image and copyright information in PMC

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