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What is Bezafibrate?

Bezafibrate, also known as Bezalip, is a drug commonly used to lower the levels of fats (lipids) in the blood, such as cholesterol. Some research studies have suggested that Bezafibrate can improve mitochondrial function within cells by increasing mitochondrial biogenesis (the process by which cells change the size and shape of their mitochondria).

How does it work?

Bezafibrate works by switching on production of PGC-1α, a protein that has an important role in mitochondrial biogenesis. There is evidence to suggest that an increase in PGC-1α can trigger mitochondrial biogenesis within cells.

What is it supposed to do?

The increase in mitochondrial biogenesis is thought to increase the amount of energy the mitochondria can produce, which might reduce some of the symptoms of mitochondrial disease.

Is it a cure?

Unfortunately not. Bezafibrate cannot correct the underlying genetic defect that causes mitochondrial disease.

What types of Mitochondrial Disease could it treat?

It is currently being trialled in patients with evidence of muscle weakness (myopathy) and a particular genetic error causing mitochondrial disease (the m.3243A>G mutation) but if successful, it could be used to treat any inherited mitochondrial disease.

Have there been any trials of Bezafibrate?

Bezafibrate has been trialled for a number of conditions (other than mitochondrial disease) and is already licensed as a treatment. Because of this, the side effects of Bezafibrate are well known. There is currently one ongoing clinical trial to study the effect of Bezafibrate in patients with mitochondrial disease. This is an open-label, non-randomised trial, meaning that both the researchers and the participants know what treatment they are taking. The study aims to assess the safety and tolerability of Bezafibrate in 10 patients with mitochondrial disease and determine its effect on mitochondrial function.

What is the latest update on this trial?

Six participants have completed the trial. Preliminary safety results show that Bezafibrate is well tolerated in patients, both at standard and higher doses. Further results are expected in late 2017.

What next?

The researchers anticipate the need for larger, randomised trials of Bezafibrate in the future. The results of the current trial will help determine how many patients would be needed in a larger trial and what measurements would be used to determine the effect of the drug.

Are there any medical papers on this trial?

Information on the current clinical trial of Bezafibrate in mitochondrial disease was presented at the 10th UK Neuromuscular Translational Research Conference. To read the conference abstract, click here

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