Characterization of a novel mitogen-activated protein kinase kinase 1/2 inhibitor with a unique mechanism of action for cancer therapy

Cancer Res. 2009 Mar 1;69(5):1924-32. doi: 10.1158/0008-5472.CAN-08-2627. Epub 2009 Feb 24.

Abstract

The mitogen-activated protein kinase (MAPK) signal transduction pathway plays a central role in regulating tumor cell growth, survival, differentiation, and angiogenesis. The key components of the Ras/Raf/MEK/ERK signal module are frequently altered in human cancers. Targeting this pathway represents a promising anticancer strategy. Small molecule inhibitors targeting MEK1/2 have shown promise in the clinic; however, ultimate clinical proof-of-concept remains elusive. Here, we report a potent and highly selective non-ATP-competitive MEK1/2 inhibitor, RO4927350, with a novel chemical structure and unique mechanism of action. It selectively blocks the MAPK pathway signaling both in vitro and in vivo, which results in significant antitumor efficacy in a broad spectrum of tumor models. Compared with previously reported MEK inhibitors, RO4927350 inhibits not only ERK1/2 but also MEK1/2 phosphorylation. In cancer cells, high basal levels of phospho-MEK1/2 rather than phospho-ERK1/2 seem to correlate with greater sensitivity to RO4927350. Furthermore, RO4927350 prevents a feedback increase in MEK phosphorylation, which has been observed with other MEK inhibitors. We show that B-Raf rather than C-Raf plays a critical role in the feedback regulation. The unique MAPK signaling blockade mediated by RO4927350 in cancer may reduce the risk of developing drug resistance. Thus, RO4927350 represents a novel therapeutic modality in cancers with aberrant MAPK pathway activation.

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology*
  • Cell Line
  • Extracellular Signal-Regulated MAP Kinases / antagonists & inhibitors
  • Extracellular Signal-Regulated MAP Kinases / metabolism
  • Female
  • Humans
  • Imidazoles / pharmacology*
  • MAP Kinase Kinase 1 / antagonists & inhibitors*
  • MAP Kinase Kinase 1 / metabolism
  • MAP Kinase Kinase 2 / antagonists & inhibitors*
  • MAP Kinase Kinase 2 / metabolism
  • MAP Kinase Signaling System / drug effects
  • Macaca fascicularis
  • Mice
  • Phosphorylation
  • Protein Kinase Inhibitors / pharmacology*
  • Thiazoles / pharmacology*

Substances

  • (2S,3S)-2-((R)-4-(4-(2-hydroxyethoxy)phenyl)-2,5-dioxo-imidazolidin-1-yl)-3-phenyl-N-(4-propionylthiazol-2-yl)butyramide
  • Antineoplastic Agents
  • Imidazoles
  • Protein Kinase Inhibitors
  • Thiazoles
  • MAP2K2 protein, human
  • Extracellular Signal-Regulated MAP Kinases
  • MAP Kinase Kinase 1
  • MAP Kinase Kinase 2
  • MAP2K1 protein, human