# Emerging Molecular Insights and Therapeutic Directions in Neurofibromatosis Type 1 and NF2-Related Schwannomatosis

## TL;DR

This paper reviews recent molecular discoveries and new treatment approaches for two genetic disorders that cause nervous system tumors.

## Contribution

The paper highlights novel therapeutic strategies and molecular insights for NF1 and NF2-related schwannomatosis.

## Key findings

- MEK inhibitors are emerging as a treatment for NF1.
- A new TβR1-RKIP pathogenic axis was discovered in NF2-related schwannomatosis.
- Brain-penetrant HDAC inhibitors show promise for NF2-SWN.

## Abstract

Neurofibromatosis type 1 (NF1) and NF2-related schwannomatosis (NF2-SWN) are major genetic tumor predisposition syndromes characterized by progressive, often debilitating neoplasms of the peripheral and central nervous systems. Over the past five years, substantial advances in molecular genetics, signaling biology, and targeted therapeutic development have reshaped diagnostic and management paradigms for both disorders. This Perspective synthesizes recent developments, including gene-based reclassification, emergence of MEK inhibitor therapy in NF1, renewed evaluation of bevacizumab and kinase-pathway inhibitor brigatinib, the discovery of a novel TβR1-RKIP pathogenic axis, and a brain-penetrant HDAC inhibitor in NF2-SWN. These insights highlight a shift toward precision-medicine strategies and mechanistically driven therapies poised to redefine future clinical care.