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Updated: Oct 10, 2025
Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens
Published on: August 25, 2023
Targeted Therapies for the Neurofibromatoses
Lauren D Sanchez1, Ashley Bui2, Laura J Klesse2
1Department of Pediatrics, Division of Neurology, UT Southwestern Medical Center, Dallas, TX 75235, USA.
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.
Insights
Management of neurofibromatosis tumors now focuses on symptom reduction. MEK inhibition shows promise for neurofibromatosis type 1, but progress for other types and malignant tumors is ongoing.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Neurofibromatosis tumors often require distinct management strategies compared to spontaneous tumors.
- Current focus is on symptom minimization due to risks of tumor persistence and growth.
Purpose of the Study:
- To review the translation of preclinical data into therapeutic trials for neurofibromatosis.
- To highlight advancements in managing neurofibromatosis type 1 and type 2.
Main Methods:
- Review of preclinical data and therapeutic trial outcomes.
- Focus on targeted therapies for specific neurofibromatosis subtypes.
Main Results:
- MEK inhibition demonstrates success in treating neurofibromatosis type 1 with progressive optic pathway gliomas or plexiform neurofibromas.
- Therapeutic success for malignant NF1 tumors (gliomas, MPNSTs) and for neurofibromatosis type 2 and schwannomatosis remains limited.
Conclusions:
- Targeted therapies, like MEK inhibitors, represent a significant advancement for specific neurofibromatosis type 1 manifestations.
- Further research is crucial for developing effective treatments for malignant NF1 tumors and for neurofibromatosis type 2 and schwannomatosis.
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09:37Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
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08:57Author Spotlight: Genetically Engineered Mouse Models and Pathological Characterization of Neurofibromatosis Type 1 Associated Tumors
Published on: May 17, 2024
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