Ribosome biogenesis as a potential therapeutic target in KRAS mutant colorectal cancer

Nat Commun. 2025 Dec 27;17(1):1224. doi: 10.1038/s41467-025-67979-9.

Abstract

Molecular targeted therapies targeting KRAS signaling have significantly improved patient outcomes, but they have not achieved sufficient therapeutic efficacy in colorectal cancer (CRC). Here, we demonstrate that a subset of KRAS-mutant CRC cells transitions to a cellular state characterized by enhanced ribosome biogenesis upon KRAS signaling inhibition. The mitogen-activated protein kinase kinase inhibitor, trametinib, and AMG510 induce a cellular state characterized by a gene expression profile highly enriched for ribosome biogenesis. We find that they are vulnerable to the inhibition of RNA polymerase I, and they exhibit synergistic anti-tumor effects with trametinib in an autochthonous mouse model of intestinal tumors and human patient-derived organoids (PDOs). These observations demonstrate that high ribosome biogenesis induced by KRAS inhibition is indispensable to maintain this cellular state and is a potential therapeutic target. Overall, this study reveals novel mechanisms of drug tolerance to KRAS inhibition, thereby facilitating the development of new therapeutic strategies.

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Colorectal Neoplasms* / drug therapy
  • Colorectal Neoplasms* / genetics
  • Colorectal Neoplasms* / metabolism
  • Colorectal Neoplasms* / pathology
  • Humans
  • Mice
  • Molecular Targeted Therapy
  • Mutation
  • Organoids / drug effects
  • Protein Kinase Inhibitors / pharmacology
  • Proto-Oncogene Proteins p21(ras)* / antagonists & inhibitors
  • Proto-Oncogene Proteins p21(ras)* / genetics
  • Proto-Oncogene Proteins p21(ras)* / metabolism
  • Pyridones / pharmacology
  • Pyridones / therapeutic use
  • Pyrimidinones / pharmacology
  • Pyrimidinones / therapeutic use
  • RNA Polymerase I / antagonists & inhibitors
  • RNA Polymerase I / metabolism
  • Ribosomes* / drug effects
  • Ribosomes* / genetics
  • Ribosomes* / metabolism
  • Signal Transduction / drug effects
  • Xenograft Model Antitumor Assays

Substances

  • Proto-Oncogene Proteins p21(ras)
  • trametinib
  • Pyridones
  • Pyrimidinones
  • KRAS protein, human
  • Protein Kinase Inhibitors
  • RNA Polymerase I