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Related Experiment Video

Updated: Oct 10, 2025

Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens

Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens

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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.

|December 10, 2021

Summary

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.

Keywords:

low grade glioma neurofibromatosis plexiform neurofibroma vestibular schwannoma

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Author Spotlight: Genetically Engineered Mouse Models and Pathological Characterization of Neurofibromatosis Type 1 Associated Tumors

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Author Spotlight: Genetically Engineered Mouse Models and Pathological Characterization of Neurofibromatosis Type 1 Associated Tumors

Author Spotlight: Genetically Engineered Mouse Models and Pathological Characterization of Neurofibromatosis Type 1 Associated Tumors

Published on: May 17, 2024

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.