Impact of Scaffold Exploration on Novel Dual-Acting Histone Deacetylases and Phosphodiesterase 5 Inhibitors for the Treatment of Alzheimer's Disease

ACS Chem Neurosci. 2017 Mar 15;8(3):638-661. doi: 10.1021/acschemneuro.6b00370. Epub 2016 Dec 27.

Abstract

A novel systems therapeutics approach, involving simultaneous inhibition of phosphodiesterase 5 (PDE5) and histone deacetylase (HDAC), has been validated as a potentially novel therapeutic strategy for the treatment of Alzheimer's disease (AD). First-in-class dual inhibitors bearing a sildenafil core have been very recently reported, and the lead molecule 7 has proven this strategy in AD animal models. Because scaffolds may play a critical role in primary activities and ADME-Tox profiling as well as on intellectual property, we have explored alternative scaffolds (vardenafil- and tadalafil-based cores) and evaluated their impact on critical parameters such as primary activities, permeability, toxicity, and in vivo (pharmacokinetics and functional response in hippocampus) to identify a potential alternative lead molecule bearing a different chemotype for in vivo testing.

Keywords: Alzheimer’s disease; HDACs; PDE5; dual inhibitor; tadalafil; vardenafil.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • 3',5'-Cyclic-AMP Phosphodiesterases / metabolism
  • Adenosine Triphosphate / metabolism
  • Alzheimer Disease / drug therapy*
  • Alzheimer Disease / pathology
  • Animals
  • Cell Line, Transformed
  • Cyclic GMP / metabolism
  • Cyclic Nucleotide Phosphodiesterases, Type 5 / metabolism
  • Histone Deacetylase Inhibitors / chemistry
  • Histone Deacetylase Inhibitors / pharmacology
  • Histone Deacetylase Inhibitors / therapeutic use*
  • Humans
  • Leukocytes, Mononuclear
  • Mice
  • Microsomes, Liver / drug effects
  • Models, Molecular
  • Neuroglia / drug effects
  • Neurons / drug effects
  • Phosphodiesterase 5 Inhibitors / chemistry
  • Phosphodiesterase 5 Inhibitors / pharmacology
  • Phosphodiesterase 5 Inhibitors / therapeutic use*

Substances

  • Histone Deacetylase Inhibitors
  • Phosphodiesterase 5 Inhibitors
  • Adenosine Triphosphate
  • 3',5'-Cyclic-AMP Phosphodiesterases
  • PDE9A protein, human
  • Cyclic Nucleotide Phosphodiesterases, Type 5
  • Cyclic GMP