Mesenchymal subtype neuroblastomas are addicted to TGF-βR2/HMGCR-driven protein geranylgeranylation

Sci Rep. 2020 Jul 1;10(1):10748. doi: 10.1038/s41598-020-67310-0.

Abstract

The identification of targeted agents with high therapeutic index is a major challenge for cancer drug discovery. We found that screening chemical libraries across neuroblastoma (NBL) tumor subtypes for selectively-lethal compounds revealed metabolic dependencies that defined each subtype. Bioactive compounds were screened across cell models of mesenchymal (MESN) and MYCN-amplified (MYCNA) NBL subtypes, which revealed the mevalonate and folate biosynthetic pathways as MESN-selective dependencies. Treatment with lovastatin, a mevalonate biosynthesis inhibitor, selectively inhibited protein prenylation and induced apoptosis in MESN cells, while having little effect in MYCNA lines. Statin sensitivity was driven by HMGCR expression, the rate-limiting enzyme for cholesterol synthesis, which correlated with statin sensitivity across NBL cell lines, thus providing a drug sensitivity biomarker. Comparing expression profiles from sensitive and resistant cell lines revealed a TGFBR2 signaling axis that regulates HMGCR, defining an actionable addiction in that leads to MESN-subtype-dependent apoptotic cell death.

Publication types

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

MeSH terms

  • Antineoplastic Agents / pharmacology
  • Apoptosis
  • Biomarkers, Tumor / metabolism
  • Cell Line, Tumor
  • Fluvastatin / pharmacology
  • Humans
  • Hydroxymethylglutaryl CoA Reductases / metabolism
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / pharmacology
  • Lipids / chemistry
  • Lovastatin / pharmacology
  • Methotrexate / pharmacology
  • N-Myc Proto-Oncogene Protein / metabolism
  • Neuroblastoma / metabolism*
  • Protein Prenylation*
  • RNA, Small Interfering / metabolism
  • Receptor, Transforming Growth Factor-beta Type II / metabolism*
  • Signal Transduction*
  • Triamterene / pharmacology

Substances

  • Antineoplastic Agents
  • Biomarkers, Tumor
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Lipids
  • MYCN protein, human
  • N-Myc Proto-Oncogene Protein
  • RNA, Small Interfering
  • Fluvastatin
  • Lovastatin
  • HMGCR protein, human
  • Hydroxymethylglutaryl CoA Reductases
  • Receptor, Transforming Growth Factor-beta Type II
  • TGFBR2 protein, human
  • Triamterene
  • Methotrexate