Deferiprone for the treatment of Friedreich's ataxia

J Neurochem. 2013 Aug;126 Suppl 1:142-6. doi: 10.1111/jnc.12300.

Abstract

Friedreich's ataxia (FRDA) is a neurological disease related to a deficiency of the protein frataxin involved in iron-sulfur (Fe-S) cluster biogenesis. This leads to an increased cellular iron uptake accumulating in mitochondria, and a subsequently disturbed iron homeostasis. The detailed mechanism of iron regulation of frataxin expression is yet unknown. Deferiprone, an iron chelator that may cross the blood-brain barrier, was shown to shuttle iron between subcellular compartments. It could also transfer iron from iron-overloaded cells to extracellular apotransferrin and pre-erythroid cells for heme synthesis. Here, clinical studies on Deferiprone are reviewed in the context of alternative agents such as desferoxamine, with specific regard to its mechanistic and clinical implications.

Keywords: Deferiprone; Friedreich's ataxia (FRDA); clinical study; frataxin.

Publication types

  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Deferiprone
  • Drug Evaluation, Preclinical
  • Friedreich Ataxia / drug therapy*
  • Humans
  • Iron / metabolism
  • Iron Chelating Agents / therapeutic use*
  • Iron-Binding Proteins / metabolism
  • Pyridones / therapeutic use*
  • Sulfur / metabolism

Substances

  • Iron Chelating Agents
  • Iron-Binding Proteins
  • Pyridones
  • frataxin
  • Deferiprone
  • Sulfur
  • Iron