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Title: Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance
Authors: Buyse, Gunnar ×
Van der Mieren, Gerry
Erb, Michael
D'hooge, Jan
Herijgers, Paul
Verbeken, Eric
Jara, Alejandro
Van Den Bergh, An
Mertens, Luc
Courdier-Fruh, Isabelle
Barzaghi, Patrizia
Meier, Thomas #
Issue Date: Jan-2009
Publisher: Elsevier
Series Title: European Heart Journal vol:30 issue:1 pages:116-124
Abstract: Aims Duchenne muscular dystrophy (DMD) is a severe and still incurable disease, with heart failure as a leading cause of death. The identification of a disease-modifying therapy may require early-initiated and long-term administration, but such type of therapeutic trial is not evident in humans. We have performed such a trial of SNT-MC17/idebenone in the mdx mouse model of DMD, based on the drug's potential to improve mitochondrial respiratory chain function and reduce oxidative stress. Methods and results In this study, 200 mg/kg bodyweight of either SNT-MC17/idebenone or placebo was given from age 4 weeks until 10 months in mdx and wild-type mice. All evaluators were blinded to mouse type and treatment groups. Idebenone treatment significantly corrected cardiac diastolic dysfunction and prevented mortality from cardiac pump failure induced by dobutamine stress testing in vivo, significantly reduced cardiac inflammation and fibrosis, and significantly improved voluntary running performance in mdx mice. Conclusion We have identified a novel potential therapeutic strategy for human DMD, as SNT-MC17/idebenone was cardioprotective and improved exercise performance in the dystrophin-deficient mdx mouse. Our data also illustrate that the mdx mouse provides unique opportunities for long-term controlled prehuman therapeutic studies.
URI: 
ISSN: 0195-668X
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Experimental Cardiac Surgery
Cardiovascular Imaging and Dynamics
Pediatric Cardiology Section (-)
Clinical Residents Medicine
Brain & Metabolism Section (-)
Translational Cell & Tissue Research
× corresponding author
# (joint) last author

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