The purpose of this study is to gather preliminary data on whether bezafibrate can improve
cellular energy production in mitochondrial disease.
Mitochondrial diseases are rare inherited disorders that arise due to deficient energy
production within the cells of the body. Consequently, the typical clinical features arise in
organs with high energy requirements. Mitochondrial disorders exhibit highly variable
clinical effects, both between individuals and within families. Characteristic symptoms
include muscle weakness (myopathy), hearing loss, migraine, epilepsy and stroke like episodes
in addition to diabetes and heart problems. Mitochondrial disorders can therefore impact
considerably on both quality of life and life expectancy. Despite this, no proven disease
modifying treatments are available.
Pre-clinical studies have identified that several existing medications improve mitochondrial
function. Of these, bezafibrate has the best supportive data and, because it is already
licensed as a treatment for high blood fats, has a well characterised side effect profile.
The investigators will therefore conduct a feasibility study of bezafibrate in people with
mitochondrial myopathy. Ten affected participants will be recruited and will receive a
titrating course of bezafibrate three times daily for 12 weeks.