A proteasome-resistant fragment of NIK mediates oncogenic NF-κB signaling in schwannomas

Hum Mol Genet. 2019 Feb 15;28(4):572-583. doi: 10.1093/hmg/ddy361.

Abstract

Schwannomas are common, highly morbid and medically untreatable tumors that can arise in patients with germ line as well as somatic mutations in neurofibromatosis type 2 (NF2). These mutations most commonly result in the loss of function of the NF2-encoded protein, Merlin. Little is known about how Merlin functions endogenously as a tumor suppressor and how its loss leads to oncogenic transformation in Schwann cells (SCs). Here, we identify nuclear factor kappa-light-chain-enhancer of activated B cells (NF-κB)-inducing kinase (NIK) as a potential drug target driving NF-κB signaling and Merlin-deficient schwannoma genesis. Using a genomic approach to profile aberrant tumor signaling pathways, we describe multiple upregulated NF-κB signaling elements in human and murine schwannomas, leading us to identify a caspase-cleaved, proteasome-resistant NIK kinase domain fragment that amplifies pathogenic NF-κB signaling. Lentiviral-mediated transduction of this NIK fragment into normal SCs promotes proliferation, survival, and adhesion while inducing schwannoma formation in a novel in vivo orthotopic transplant model. Furthermore, we describe an NF-κB-potentiated hepatocyte growth factor (HGF) to MET proto-oncogene receptor tyrosine kinase (c-Met) autocrine feed-forward loop promoting SC proliferation. These innovative studies identify a novel signaling axis underlying schwannoma formation, revealing new and potentially druggable schwannoma vulnerabilities with future therapeutic potential.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Autocrine Communication / genetics
  • Carcinogenesis / genetics
  • Caspase 1 / genetics
  • Cell Proliferation / genetics
  • Disease Models, Animal
  • Gene Expression Regulation, Neoplastic
  • Hepatocyte Growth Factor / genetics
  • Humans
  • Mice
  • Molecular Targeted Therapy
  • NF-kappa B / genetics
  • NF-kappaB-Inducing Kinase
  • Neurilemmoma / complications
  • Neurilemmoma / drug therapy
  • Neurilemmoma / genetics*
  • Neurilemmoma / pathology
  • Neurofibromatosis 2 / complications
  • Neurofibromatosis 2 / drug therapy
  • Neurofibromatosis 2 / genetics*
  • Neurofibromatosis 2 / pathology
  • Neurofibromin 2 / genetics*
  • Proteasome Endopeptidase Complex / genetics
  • Protein Serine-Threonine Kinases / genetics*
  • Proto-Oncogene Mas
  • Proto-Oncogene Proteins c-met / genetics
  • Schwann Cells
  • Signal Transduction / genetics

Substances

  • HGF protein, human
  • MAS1 protein, human
  • NF-kappa B
  • Neurofibromin 2
  • Proto-Oncogene Mas
  • Hepatocyte Growth Factor
  • Proto-Oncogene Proteins c-met
  • Protein Serine-Threonine Kinases
  • Caspase 1
  • Proteasome Endopeptidase Complex