Activation of tumor suppressor protein PP2A inhibits KRAS-driven tumor growth

J Clin Invest. 2017 Jun 1;127(6):2081-2090. doi: 10.1172/JCI89548. Epub 2017 May 15.

Abstract

Targeted cancer therapies, which act on specific cancer-associated molecular targets, are predominantly inhibitors of oncogenic kinases. While these drugs have achieved some clinical success, the inactivation of kinase signaling via stimulation of endogenous phosphatases has received minimal attention as an alternative targeted approach. Here, we have demonstrated that activation of the tumor suppressor protein phosphatase 2A (PP2A), a negative regulator of multiple oncogenic signaling proteins, is a promising therapeutic approach for the treatment of cancers. Our group previously developed a series of orally bioavailable small molecule activators of PP2A, termed SMAPs. We now report that SMAP treatment inhibited the growth of KRAS-mutant lung cancers in mouse xenografts and transgenic models. Mechanistically, we found that SMAPs act by binding to the PP2A Aα scaffold subunit to drive conformational changes in PP2A. These results show that PP2A can be activated in cancer cells to inhibit proliferation. Our strategy of reactivating endogenous PP2A may be applicable to the treatment of other diseases and represents an advancement toward the development of small molecule activators of tumor suppressor proteins.

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology*
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Drug Resistance, Neoplasm
  • Enzyme Activation
  • Enzyme Activators / chemistry
  • Enzyme Activators / pharmacology*
  • Humans
  • Male
  • Mice, Inbred BALB C
  • Mice, Nude
  • Mice, Transgenic
  • Protein Binding
  • Protein Phosphatase 2 / chemistry
  • Protein Phosphatase 2 / metabolism*
  • Proto-Oncogene Proteins p21(ras) / genetics*
  • Signal Transduction
  • Tumor Burden
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Enzyme Activators
  • KRAS protein, human
  • Protein Phosphatase 2
  • Proto-Oncogene Proteins p21(ras)