Tasquinimod Is an Allosteric Modulator of HDAC4 survival signaling within the compromised cancer microenvironment

Cancer Res. 2013 Feb 15;73(4):1386-99. doi: 10.1158/0008-5472.CAN-12-2730. Epub 2012 Nov 13.

Abstract

Tasquinimod is an orally active antiangiogenic drug that is currently in phase III clinical trials for the treatment of castration-resistant prostate cancer. However, the target of this drug has remained unclear. In this study, we applied diverse strategies to identify the histone deacetylase HDAC4 as a target for the antiangiogenic activity of tasquinimod. Our comprehensive analysis revealed allosteric binding (Kd 10-30 nmol/L) to the regulatory Zn(2+) binding domain of HDAC4 that locks the protein in a conformation preventing HDAC4/N-CoR/HDAC3 complex formation. This binding inhibited colocalization of N-CoR/HDAC3, thereby inhibiting deacetylation of histones and HDAC4 client transcription factors, such as HIF-1α, which are bound at promoter/enhancers where epigenetic reprogramming is required for cancer cell survival and angiogenic response. Through this mechanism, tasquinimod is effective as a monotherapeutic agent against human prostate, breast, bladder, and colon tumor xenografts, where its efficacy could be further enhanced in combination with a targeted thapsigargin prodrug (G202) that selectively kills tumor endothelial cells. Together, our findings define a mechanism of action of tasquinimod and offer a perspective on how its clinical activity might be leveraged in combination with other drugs that target the tumor microenvironment. Cancer Res; 73(4); 1386-99. ©2012 AACR.

Publication types

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

MeSH terms

  • Acetylation / drug effects
  • Allosteric Regulation
  • Animals
  • Blotting, Western
  • Cell Hypoxia
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Histone Deacetylases / chemistry
  • Histone Deacetylases / genetics
  • Histone Deacetylases / metabolism*
  • Histones / metabolism
  • Human Umbilical Vein Endothelial Cells / drug effects
  • Human Umbilical Vein Endothelial Cells / metabolism
  • Humans
  • Hypoxia-Inducible Factor 1, alpha Subunit / genetics
  • Hypoxia-Inducible Factor 1, alpha Subunit / metabolism
  • Mice
  • Mice, Nude
  • Models, Molecular
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / pathology
  • Prodrugs / pharmacology
  • Protein Binding / drug effects
  • Protein Structure, Tertiary
  • Quinolines / pharmacology*
  • Quinolones
  • RNA Interference
  • Repressor Proteins / chemistry
  • Repressor Proteins / genetics
  • Repressor Proteins / metabolism*
  • Signal Transduction / drug effects*
  • Thapsigargin / pharmacology
  • Tumor Microenvironment / drug effects*
  • Tumor Microenvironment / genetics
  • Xenograft Model Antitumor Assays*

Substances

  • HIF1A protein, human
  • Histones
  • Hypoxia-Inducible Factor 1, alpha Subunit
  • Prodrugs
  • Quinolines
  • Quinolones
  • Repressor Proteins
  • Thapsigargin
  • tasquinimod
  • HDAC4 protein, human
  • Histone Deacetylases