Ironing out the Brain - is Chelation a Therapeutic Option

Title: Ironing out the Brain - is Chelation a Therapeutic Option

Speaker: Robert R. Crichton

Afillitation: Universite Catholique de Louvain, Louvain-la-Neuve, Belgium

Abstract: While iron is an essential element for almost all living organisms, it has a darker side, namely its toxicity. This is particularly the case for humans, exacerbated by their limited capacity to excrete iron. Both anaemia, due to dietary deficiency, inflammation, infection or genetic aberration, and iron overload, either hereditary haemochromatosis due to mutations in proteins involved in iron homeostasis or secondary haemochromatosis due to genetic dysfunction of erythropoiesis, are common. To treat patients with secondary haemochromatosis, the iron overload resulting from chronic blood transfusions is removed by the administration of iron chelators, and in this presentation we will briefly outline the development of these iron chelators starting with the bacterial siderophore desferrioxamine and leading to the orally active synthetic chelators deferiprone and deferasirox.

However, it has become clear that many neurodegenerative diseases are characterised by iron accumulation in specific brain regions, including Parkinson’s disease, Alzheimer’s disease, Huntington’s disease and Friedreich’s ataxia, and the potential for iron chelation as a therapeutic option is beginning to be actively investigated. In the second part of the presentation we will outline the results from recent clinical trials to evaluate the use of deferiprone in patients with Parkinson’s disease and draw conclusions regarding the future use of iron chelation therapy in the treatment of neurodegenerative diseases.