Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease

Abstract Parkinson’s disease (PD) is associated with increased iron levels in the substantia nigra (SNc). This study evaluated whether the iron chelator, deferiprone, is well tolerated, able to chelate iron from various brain regions and improve PD symptomology. In a randomised double-blind, placebo...

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Autores principales:	Antonio Martin-Bastida, Roberta J. Ward, Rexford Newbould, Paola Piccini, David Sharp, Christina Kabba, Maneesh C. Patel, Michael Spino, John Connelly, Fernando Tricta, Robert R. Crichton, David T. Dexter
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2017
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/447847d27a3f44eea2861e4c6d11074c
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spelling	oai:doaj.org-article:447847d27a3f44eea2861e4c6d11074c2021-12-02T15:06:15ZBrain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease10.1038/s41598-017-01402-22045-2322https://doaj.org/article/447847d27a3f44eea2861e4c6d11074c2017-05-01T00:00:00Zhttps://doi.org/10.1038/s41598-017-01402-2https://doaj.org/toc/2045-2322Abstract Parkinson’s disease (PD) is associated with increased iron levels in the substantia nigra (SNc). This study evaluated whether the iron chelator, deferiprone, is well tolerated, able to chelate iron from various brain regions and improve PD symptomology. In a randomised double-blind, placebo controlled trial, 22 early onset PD patients, were administered deferiprone, 10 or 15 mg/kg BID or placebo, for 6 months. Patients were evaluated for PD severity, cognitive function, depression rating and quality of life. Iron concentrations were assessed in the substantia nigra (SNc), dentate and caudate nucleus, red nucleus, putamen and globus pallidus by T2* MRI at baseline and after 3 and 6 months of treatment. Deferiprone therapy was well tolerated and was associated with a reduced dentate and caudate nucleus iron content compared to placebo. Reductions in iron content of the SNc occurred in only 3 patients, with no changes being detected in the putamen or globus pallidus. Although 30 mg/kg deferiprone treated patients showed a trend for improvement in motor-UPDRS scores and quality of life, this did not reach significance. Cognitive function and mood were not adversely affected by deferiprone therapy. Such data supports more extensive clinical trials into the potential benefits of iron chelation in PD.Antonio Martin-BastidaRoberta J. WardRexford NewbouldPaola PicciniDavid SharpChristina KabbaManeesh C. PatelMichael SpinoJohn ConnellyFernando TrictaRobert R. CrichtonDavid T. DexterNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 7, Iss 1, Pp 1-9 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Antonio Martin-Bastida Roberta J. Ward Rexford Newbould Paola Piccini David Sharp Christina Kabba Maneesh C. Patel Michael Spino John Connelly Fernando Tricta Robert R. Crichton David T. Dexter Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease
description	Abstract Parkinson’s disease (PD) is associated with increased iron levels in the substantia nigra (SNc). This study evaluated whether the iron chelator, deferiprone, is well tolerated, able to chelate iron from various brain regions and improve PD symptomology. In a randomised double-blind, placebo controlled trial, 22 early onset PD patients, were administered deferiprone, 10 or 15 mg/kg BID or placebo, for 6 months. Patients were evaluated for PD severity, cognitive function, depression rating and quality of life. Iron concentrations were assessed in the substantia nigra (SNc), dentate and caudate nucleus, red nucleus, putamen and globus pallidus by T2* MRI at baseline and after 3 and 6 months of treatment. Deferiprone therapy was well tolerated and was associated with a reduced dentate and caudate nucleus iron content compared to placebo. Reductions in iron content of the SNc occurred in only 3 patients, with no changes being detected in the putamen or globus pallidus. Although 30 mg/kg deferiprone treated patients showed a trend for improvement in motor-UPDRS scores and quality of life, this did not reach significance. Cognitive function and mood were not adversely affected by deferiprone therapy. Such data supports more extensive clinical trials into the potential benefits of iron chelation in PD.
format	article
author	Antonio Martin-Bastida Roberta J. Ward Rexford Newbould Paola Piccini David Sharp Christina Kabba Maneesh C. Patel Michael Spino John Connelly Fernando Tricta Robert R. Crichton David T. Dexter
author_facet	Antonio Martin-Bastida Roberta J. Ward Rexford Newbould Paola Piccini David Sharp Christina Kabba Maneesh C. Patel Michael Spino John Connelly Fernando Tricta Robert R. Crichton David T. Dexter
author_sort	Antonio Martin-Bastida
title	Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease
title_short	Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease
title_full	Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease
title_fullStr	Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease
title_full_unstemmed	Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease
title_sort	brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in parkinson’s disease
publisher	Nature Portfolio
publishDate	2017
url	https://doaj.org/article/447847d27a3f44eea2861e4c6d11074c
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Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease

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