THERAPEUTIC EFFECT OF IRON CHELATOR ON FRIEDREICH’S ATAXIA

Abstract

Friedreich's ataxia (FRDA) is an inherited neurodegenerative disease caused by reduced levels of the mitochondrial protein frataxin. The loss of frataxin leads to the accumulation of mitochondrial iron ions and causes cell damage. The literature review will provide an overview of the mechanisms underlying the FRDA disease leading to mitochondrial iron overload, the present therapies and future approaches with potential for the therapy of the disease. Notably, through analysis of the pathogenesis and the results of clinical trials of existing iron chelating agents the review will concentrate on iron chelation as a potential therapy for FRDA. While iron chelation therapy (ICT) has shown some benefit to FRDA patients, the non-specific distribution of iron chelators throughout the body promotes toxicity. Therefore, ICT with iron chelating agents that are targeted to mitochondria may be a better choice to specifically sequester the excess free iron in the organelle. This can provide both neuro- and cardio-protection to FRDA patients, reinstating their body’s defective functions.