Cellular Origin, Tumor Progression, and Pathogenic Mechanisms of Cutaneous Neurofibromas Revealed by Mice with Nf1 Knockout in Boundary Cap Cells

Cancer Discov. 2019 Jan;9(1):130-147. doi: 10.1158/2159-8290.CD-18-0156. Epub 2018 Oct 22.

Abstract

Patients carrying an inactive NF1 allele develop tumors of Schwann cell origin called neurofibromas (NF). Genetically engineered mouse models have significantly enriched our understanding of plexiform forms of NFs (pNF). However, this has not been the case for cutaneous neurofibromas (cNF), observed in all NF1 patients, as no previous model recapitulates their development. Here, we show that conditional Nf1 inactivation in Prss56-positive boundary cap cells leads to bona fide pNFs and cNFs. This work identifies subepidermal glia as a likely candidate for the cellular origin of cNFs and provides insights on disease mechanisms, revealing a long, multistep pathologic process in which inflammation-related signals play a pivotal role. This new mouse model is an important asset for future clinical and therapeutic investigations of NF1-associated neurofibromas. SIGNIFICANCE: Patients affected by NF1 develop numerous cNFs. We present a mouse model that faithfully recapitulates cNFs, identify a candidate cell type at their origin, analyze the steps involved in their formation, and show that their development is dramatically accelerated by skin injury. These findings have important clinical/therapeutic implications.This article is highlighted in the In This Issue feature, p. 1.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Disease Models, Animal
  • Female
  • Male
  • Mice
  • Mice, Knockout
  • Mutation
  • Neurofibroma / etiology
  • Neurofibroma / genetics
  • Neurofibroma / metabolism*
  • Neurofibroma / physiopathology
  • Neurofibromatosis 1 / complications
  • Neurofibromatosis 1 / genetics
  • Neurofibromatosis 1 / metabolism*
  • Neurofibromatosis 1 / physiopathology
  • Neurofibromin 1 / genetics*
  • Schwann Cells / metabolism*
  • Schwann Cells / physiology
  • Skin Neoplasms / etiology
  • Skin Neoplasms / genetics
  • Skin Neoplasms / metabolism*
  • Skin Neoplasms / physiopathology

Substances

  • Neurofibromin 1