Deferiprone for the treatment of Friedreich's ataxia.

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.

• Authors: Massimo Pandolfo, Laura Hausmann
• Journal: Journal of neurochemistry (J Neurochem) Vol. 126 Suppl 1 Pg. 142-6 (Aug 2013) ISSN: 1471-4159 [Electronic] England
• PMID: 23859349 (Publication Type: Journal Article, Review)
• Copyright: © 2013 International Society for Neurochemistry.

Chemical References

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

Topics

• 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)