Mitochondrial disorders and drugs: what every physician should know

Article Details

Authors
Daniele Orsucci MD, Elena Caldarazzo Ienco MD, Gabriele Siciliano MD, PhD, Michelangelo Mancuso MD, PhD

Article Type
Review

DOI
10.7573/dic.212588

Related Articles

Article Page

Abstract

Mitochondrial disorders are a group of metabolic conditions caused by impairment of the oxidative phosphorylation system. There is currently no clear evidence supporting any pharmacological interventions for most mitochondrial disorders, except for coenzyme Q10 deficiencies, Leber hereditary optic neuropathy, and mitochondrial neurogastrointestinal encephalomyopathy. Furthermore, some drugs may potentially have detrimental effects on mitochondrial dysfunction. Drugs known to be toxic for mitochondrial functions should be avoided whenever possible. Mitochondrial patients needing one of these treatments should be carefully monitored, clinically and by laboratory exams, including creatine kinase and lactate. In the era of molecular and ‘personalized’ medicine, many different physicians (not only neurologists) should be aware of the basic principles of mitochondrial medicine and its therapeutic implications. Multicenter collaboration is essential for the advancement of therapy for mitochondrial disorders. Whenever possible, randomized clinical trials are necessary to establish efficacy and safety of drugs. In this review we discuss in an accessible way the therapeutic approaches and perspectives in mitochondrial disorders. We will also provide an overview of the drugs that should be used with caution in these patients.

Keywords: coenzyme Q10, drugs, mitochondria, mtDNA, mitochondrial diseases, toxicity

Citation: Orsucci D, Caldarazzo Ienco E, Siciliano G, Mancuso M. Mitochondrial disorders and drugs: what every physician should know. Drugs in Context 2019; 8: 212588. DOI: 10.7573/dic.212588

Contributions: All authors contributed to the preparation of this review. All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.

Disclosure and potential conflicts of interest: The authors declare that they have no conflicts of interest. The International Committee of Medical Journal Editors (ICMJE) Potential Conflicts of Interests form for the authors is available for download at https://www.drugsincontext.com/wp-content/uploads/2019/06/dic.212588-COI.pdf

Acknowledgments: None.

Funding declaration: This work was partially supported by Telethon Grant GUP09004 and Telethon-MITOCON grant GSP16001.

Copyright: Copyright © 2019 Orsucci D, Caldarazzo Ienco E, Siciliano G, Mancuso M. https://doi.org/10.7573/dic.212588. Published by Drugs in
Context under Creative Commons License Deed CC BY NC ND 4.0 which allows anyone to copy, distribute, and transmit the article provided it is
properly attributed in the manner specified below. No commercial use without permission.

Correct attribution: Copyright © 2019 Orsucci D, Caldarazzo Ienco E, Siciliano G, Mancuso M. Published by Drugs in Context under Creative Commons License Deed CC BY NC ND 4.0.

Article URL: https://www.drugsincontext.com/mitochondrial-disorders-and-drugs:-what-every-physician-should-know/

Correspondence: Daniele Orsucci, MD, Unit of Neurology, San Luca Hospital of Lucca, Via Lippi-Francesconi, 55100 Lucca, Italy. orsuccid@gmail.com

Provenance: invited; externally peer reviewed.

Submitted: 18 March 2019; Peer review comments to author: 17 May 2019; Revised manuscript received: 30 May 2019; Accepted: 3 June 2019; Publication date: 4 July 2019.

Drugs in Context is published by BioExcel Publishing Ltd. Registered office: Plaza Building, Lee High Road, London, England, SE13 5PT.

BioExcel Publishing Limited is registered in England Number 10038393. VAT GB 252 7720 07.

For all manuscript and submissions enquiries, contact the Editor-in-Chief gordon.mallarkey@bioexcelpublishing.com

For all permissions, rights and reprints, contact David Hughes david.hughes@bioexcelpublishing.com

Download free full text PDF