Protective efficacy of phosphodiesterase-1 inhibition against alpha-synuclein toxicity revealed by compound screening in LUHMES cells

Sci Rep. 2017 Sep 13;7(1):11469. doi: 10.1038/s41598-017-11664-5.

Abstract

α-synuclein-induced neurotoxicity is a core pathogenic event in neurodegenerative synucleinopathies such as Parkinson's disease, dementia with Lewy bodies, or multiple system atrophy. There is currently no disease-modifying therapy available for these diseases. We screened 1,600 FDA-approved drugs for their efficacy to protect LUHMES cells from degeneration induced by wild-type α-synuclein and identified dipyridamole, a non-selective phosphodiesterase inhibitor, as top hit. Systematic analysis of other phosphodiesterase inhibitors identified a specific phosphodiesterase 1 inhibitor as most potent to rescue from α-synuclein toxicity. Protection was mediated by an increase of cGMP and associated with the reduction of a specific α-synuclein oligomeric species. RNA interference experiments confirmed PDE1A and to a smaller extent PDE1C as molecular targets accounting for the protective efficacy. PDE1 inhibition also rescued dopaminergic neurons from wild-type α-synuclein induced degeneration in the substantia nigra of mice. In conclusion, this work identifies inhibition of PDE1A in particular as promising target for neuroprotective treatment of synucleinopathies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line
  • Dipyridamole / pharmacology
  • Drug Discovery*
  • Drug Evaluation, Preclinical
  • Enzyme Inhibitors / pharmacology*
  • High-Throughput Screening Assays
  • Humans
  • Mice
  • Neurons / drug effects
  • Neurons / metabolism
  • Neuroprotective Agents / pharmacology
  • Phosphodiesterase I / antagonists & inhibitors*
  • Protein Aggregation, Pathological / drug therapy
  • Protein Aggregation, Pathological / metabolism*
  • Vinca Alkaloids / pharmacology
  • alpha-Synuclein / antagonists & inhibitors
  • alpha-Synuclein / metabolism*

Substances

  • Enzyme Inhibitors
  • Neuroprotective Agents
  • Vinca Alkaloids
  • alpha-Synuclein
  • vinpocetine
  • Dipyridamole
  • Phosphodiesterase I