Copper(II) Binding to PBT2 Differs from That of Other 8-Hydroxyquinoline Chelators: Implications for the Treatment of Neurodegenerative Protein Misfolding Diseases

Inorg Chem. 2020 Dec 7;59(23):17519-17534. doi: 10.1021/acs.inorgchem.0c02754. Epub 2020 Nov 23.

Abstract

PBT2 (5,7-dichloro-2-[(dimethylamino)methyl]-8-hydroxyquinoline) is a small Cu(II)-binding drug that has been investigated in the treatment of neurodegenerative diseases, namely, Alzheimer's disease (AD). PBT2 is thought to be highly effective at crossing the blood-brain barrier and has been proposed to exert anti-Alzheimer's effects through the modulation of metal ion concentrations in the brain, specifically the sequestration of Cu(II) from amyloid plaques. However, despite promising initial results in animal models and in clinical trials where PBT2 was shown to improve cognitive function, larger-scale clinical trials did not find PBT2 to have a significant effect on the amyloid plaque burden compared with controls. We propose that the results of these clinical trials likely point to a more complex mechanism of action for PBT2 other than simple Cu(II) sequestration. To this end, herein we have investigated the solution chemistry of Cu(II) coordination by PBT2 primarily using X-ray absorption spectroscopy (XAS), high-energy-resolution fluorescence-detected XAS, and electron paramagnetic resonance. We propose that a novel bis-PBT2 Cu(II) complex with asymmetric coordination may coexist in solution with a symmetric four-coordinate Cu(II)-bis-PBT2 complex distorted from coplanarity. Additionally, PBT2 is a more flexible ligand than other 8HQs because it can act as both a bidentate and a tridentate ligand as well as coordinate Cu(II) in both 1:1 and 2:1 PBT2/Cu(II) complexes.

MeSH terms

  • Alzheimer Disease / drug therapy*
  • Animals
  • Chelating Agents / chemical synthesis
  • Chelating Agents / chemistry
  • Chelating Agents / therapeutic use*
  • Clioquinol / analogs & derivatives*
  • Clioquinol / chemistry
  • Clioquinol / therapeutic use
  • Coordination Complexes / chemical synthesis
  • Coordination Complexes / chemistry
  • Coordination Complexes / therapeutic use*
  • Copper / chemistry
  • Copper / therapeutic use*
  • Density Functional Theory
  • Humans
  • Ligands
  • Molecular Structure
  • Neuroprotective Agents / chemical synthesis
  • Neuroprotective Agents / chemistry
  • Neuroprotective Agents / pharmacology*
  • Proteostasis Deficiencies / drug therapy*
  • X-Ray Absorption Spectroscopy

Substances

  • Chelating Agents
  • Coordination Complexes
  • Ligands
  • Neuroprotective Agents
  • PBT2 compound
  • Copper
  • Clioquinol