Design and synthesis of orally bioavailable aminopyrrolidinone histone deacetylase 6 inhibitors

J Med Chem. 2015 Mar 26;58(6):2809-20. doi: 10.1021/jm502011f. Epub 2015 Mar 13.

Abstract

Histone deacetylase 6 (HDAC6) removes the acetyl group from lysine residues in a number of non-histone substrates and plays important roles in microtubule dynamics and chaperone activities. There is growing interest in identifying HDAC6-selective inhibitors as chemical biology tools and ultimately as new therapeutic agents. Herein we report the design, synthesis, and phenotypic screening of a novel class of 3-aminopyrrolidinone-based hydroxamic acids as HDAC6 inhibitors. In particular, the α-methyl-substituted enantiomer 33 (3-S) showed significant in-cell tubulin acetylation (Tub-Ac) with an EC50 of 0.30 μM but limited impact on p21 levels at various concentrations. In enzyme inhibition assays, 33 demonstrated high selectivity for HDAC6 with an IC50 of 0.017 μM and selectivity indexes of 10 against HDAC8 and over 4000 against HDAC1-3 isoforms. Moreover, 33 has suitable drug metabolism and pharmacokinetics properties compared with other hydroxamic acid-based HDAC inhibitors, warranting further biological studies and development as a selective HDAC6 inhibitor.

MeSH terms

  • Administration, Oral
  • Animals
  • Cell Line
  • Drug Design
  • Histone Deacetylase 6
  • Histone Deacetylase Inhibitors / administration & dosage
  • Histone Deacetylase Inhibitors / chemistry*
  • Histone Deacetylase Inhibitors / pharmacokinetics*
  • Histone Deacetylase Inhibitors / pharmacology
  • Histone Deacetylases / metabolism*
  • Humans
  • Hydroxamic Acids / administration & dosage
  • Hydroxamic Acids / chemistry*
  • Hydroxamic Acids / pharmacokinetics*
  • Hydroxamic Acids / pharmacology
  • Male
  • Mice
  • Models, Molecular
  • Protein Isoforms / metabolism
  • Pyrrolidinones / administration & dosage
  • Pyrrolidinones / chemistry*
  • Pyrrolidinones / pharmacokinetics*
  • Pyrrolidinones / pharmacology

Substances

  • Histone Deacetylase Inhibitors
  • Hydroxamic Acids
  • Protein Isoforms
  • Pyrrolidinones
  • HDAC6 protein, human
  • Histone Deacetylase 6
  • Histone Deacetylases