Ceruloplasmin dysfunction and therapeutic potential for Parkinson disease

Ann Neurol. 2013 Apr;73(4):554-9. doi: 10.1002/ana.23817. Epub 2013 Feb 19.

Abstract

Ceruloplasmin is an iron-export ferroxidase that is abundant in plasma and also expressed in glia. We found a ∼80% loss of ceruloplasmin ferroxidase activity in the substantia nigra of idiopathic Parkinson disease (PD) cases, which could contribute to the pro-oxidant iron accumulation that characterizes the pathology. Consistent with a role for ceruloplasmin in PD etiopathogenesis, ceruloplasmin knockout mice developed parkinsonism that was rescued by iron chelation. Additionally, peripheral infusion of ceruloplasmin attenuated neurodegeneration and nigral iron elevation in the 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine mouse model for PD. These findings show, in principle, that intravenous ceruloplasmin may have therapeutic potential in PD.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine
  • Aged
  • Animals
  • Case-Control Studies
  • Ceruloplasmin / deficiency
  • Ceruloplasmin / metabolism*
  • Ceruloplasmin / therapeutic use
  • Deferiprone
  • Disease Models, Animal
  • Dopamine Agents
  • Female
  • Humans
  • Iron Chelating Agents / therapeutic use
  • Male
  • Mice
  • Mice, Knockout
  • Parkinson Disease / drug therapy*
  • Parkinson Disease / etiology
  • Parkinson Disease / metabolism*
  • Parkinson Disease / pathology
  • Pyridones / therapeutic use
  • Substantia Nigra / metabolism

Substances

  • Dopamine Agents
  • Iron Chelating Agents
  • Pyridones
  • Deferiprone
  • 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine
  • Ceruloplasmin