Simvastatin does not improve the pathology of Duchenne muscular dystrophy (DMD), a study using a mouse model of the disease found. Therefore, it does not seem to be a therapeutic option for patients with DMD, according to the authors of the study published in Skeletal Muscle.
Statins have been suggested to have beneficial effects in the mdx mouse, a popular model used to study DMD. For example, a study conducted in 2015 found that simvastatin could improve muscle health, reduce inflammation and oxidative stress, and increase autophagy in the mouse model of the disease. However, other studies have shown that statins may actually induce skeletal muscle myopathy.
To investigate whether simvastatin could really be a potential therapy for patients with DMD, a team of researchers led by Agnieszka Łoboda, PhD, DSc, conducted a number of experiments to study simvastatin-related changes in the mdx mouse. These included measuring the animals’ grip strength to assess their muscle function, treadmill tests, single-muscle force estimations, enzymatic assays, and muscle damage and gene expression analyses.
The results showed that treating the mdx mice with simvastatin did not improve running performance, grip strength, or specific force of the single muscle.
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When they checked the creatine kinase levels and lactate dehydrogenase activity in the animals, both markers of muscle injury, the researchers also observed no differences following simvastatin treatment.
Finally, there were no significant changes in inflammation, fibrosis, or angiogenesis following simvastatin treatment, nor were there any changes in regeneration-related parameters apart from a lower percentage of centrally nucleated myofibers in the gastrocnemius muscle.
“Of note, even an increased rate of necrosis was found in simvastatin-treated mdx mice,” the researchers wrote. They concluded that simvastatin does not have a significant influence on DMD pathology in the mouse model of the disease and that their results do not support the hypothesis that it could be a potential treatment for DMD.
Mucha O, Podkalicka P, Kaziród K, Samborowska E, Dulak J, Łoboda A. Simvastatin does not alleviate muscle pathology in a mouse model of Duchenne muscular dystrophy. Skelet Muscle. 2021;11(1):21. doi:10.1186/s13395-021-00276-3