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Treatment of Mitochondrial Disorders

  • PEDIATRIC NEUROLOGY (HS SINGER, SECTION EDITOR)
  • Published:
Current Treatment Options in Neurology Aims and scope Submit manuscript

Opinion statement

While numerous treatments for mitochondrial disorders have been suggested, relatively few have undergone controlled clinical trials. Treatment of these disorders is challenging, as only symptomatic therapy is available. In this review we will focus on newer drugs and treatment trials in mitochondrial diseases, with a special focus on medications to avoid in treating epilepsy and ICU patient with mitochondrial disease, which has not been included in such a review. Readers are also referred to the opinion statement in A Modern Approach to the Treatment of Mitochondrial Disease published in Current Treatment Options in Neurology 2009. Many of the supplements used for treatment were reviewed in the previous abstract, and dosing guidelines were provided. The focus of this review is on items not previously covered in depth, and our discussion includes more recently studied compounds as well as any relevant updates on older compounds . We review a variety of vitamins and xenobiotics, including dichloroacetate (DCA), arginine, coenzyme Q10, idebenone, EPI-743, and exercise training. Treatment of epilepsy, which is a common feature in many mitochondrial phenotypes, warrants special consideration due to the added toxicity of certain medications, and we provide a discussion of these unique treatment challenges. Interesting, however, with only a few exceptions, the treatment strategies for epilepsy in mitochondrial cytopathies are the same as for epilepsy without mitochondrial dysfunction. We also discuss intensive care management, building upon similar reviews, adding new dimensions, and demonstrating the complexity of overall care of these patients.

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Conflict of Interest

Sumit Parikh has served on boards for the United Mitochondrial Disease Foundation, the International Foundation for CDKL5 Research, and the Cyclic Vomiting Syndrome Association; has served as a consultant for GeneDx Laboratories; has received grant support from the NIH/NAMDC; and has served on the speakers bureau for UMDF.

Andrea Gropman has received grant support from the North American Mitochondrial Consortium and has received payment for development of educational presentations from Shire.

Sreenivas Avula, Scott Demarest, and Jonathan Kurz declare that they have no conflict of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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Authors and Affiliations

  1. Department of Neurology, Cleveland Clinic, Cleveland, OH, USA

    Sreenivas Avula MD & Sumit Parikh MD

  2. Department of Neurology, Children’s National Medical Center and the George Washington University of the Health Sciences, 111 Michigan Avenue NW, Washington, DC, 20010, USA

    Scott Demarest MD, Jonathan Kurz MD & Andrea Gropman MD

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  1. Sreenivas Avula MD
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  2. Sumit Parikh MD
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Correspondence to Andrea Gropman MD.

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This article is part of the Topical Collection on Pediatric Neurology

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Avula, S., Parikh, S., Demarest, S. et al. Treatment of Mitochondrial Disorders. Curr Treat Options Neurol 16, 292 (2014). https://doi.org/10.1007/s11940-014-0292-7

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