Tetrahydroquinoline-Capped Histone Deacetylase 6 Inhibitor SW-101 Ameliorates Pathological Phenotypes in a Charcot-Marie-Tooth Type 2A Mouse Model

J Med Chem. 2021 Apr 22;64(8):4810-4840. doi: 10.1021/acs.jmedchem.0c02210. Epub 2021 Apr 8.

Abstract

Histone deacetylase 6 (HDAC6) is a promising therapeutic target for the treatment of neurodegenerative disorders. SW-100 (1a), a phenylhydroxamate-based HDAC6 inhibitor (HDAC6i) bearing a tetrahydroquinoline (THQ) capping group, is a highly potent and selective HDAC6i that was shown to be effective in mouse models of Fragile X syndrome and Charcot-Marie-Tooth disease type 2A (CMT2A). In this study, we report the discovery of a new THQ-capped HDAC6i, termed SW-101 (1s), that possesses excellent HDAC6 potency and selectivity, together with markedly improved metabolic stability and druglike properties compared to SW-100 (1a). X-ray crystallography data reveal the molecular basis of HDAC6 inhibition by SW-101 (1s). Importantly, we demonstrate that SW-101 (1s) treatment elevates the impaired level of acetylated α-tubulin in the distal sciatic nerve, counteracts progressive motor dysfunction, and ameliorates neuropathic symptoms in a CMT2A mouse model bearing mutant MFN2. Taken together, these results bode well for the further development of SW-101 (1s) as a disease-modifying HDAC6i.

Publication types

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

MeSH terms

  • Acetylation
  • Animals
  • Benzamides / chemistry
  • Benzamides / metabolism
  • Binding Sites
  • Charcot-Marie-Tooth Disease / drug therapy*
  • Charcot-Marie-Tooth Disease / metabolism
  • Charcot-Marie-Tooth Disease / pathology
  • Crystallography, X-Ray
  • Disease Models, Animal
  • Half-Life
  • Histone Deacetylase 6 / antagonists & inhibitors*
  • Histone Deacetylase 6 / metabolism
  • Histone Deacetylase Inhibitors / chemistry
  • Histone Deacetylase Inhibitors / metabolism
  • Histone Deacetylase Inhibitors / therapeutic use*
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Molecular Docking Simulation
  • Phenotype
  • Protein Isoforms / antagonists & inhibitors
  • Protein Isoforms / metabolism
  • Quinolines / chemistry*
  • Quinolines / metabolism
  • Quinolines / therapeutic use
  • Structure-Activity Relationship
  • Tubulin / metabolism

Substances

  • Benzamides
  • Histone Deacetylase Inhibitors
  • Protein Isoforms
  • Quinolines
  • SW-100
  • Tubulin
  • 1,2,3,4-tetrahydroquinoline
  • Histone Deacetylase 6

Supplementary concepts

  • Charcot-Marie-Tooth disease, Type 2A