Mito research around the world


What is Bezafibrate?

Bezafibrate, also known as Bezalip or Befizal, 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?

Bezafibrate has been trialed in patients with evidence of muscle weakness (myopathy) and a particular genetic error causing mitochondrial disease (the m.3243A>G mutation). It is currently being trialed as a treatment for Leber’s Hereditary Optic Neuropathy (LHON), a mitochondrial disease in which symptoms are almost uniquely limited to the cells at the back of the eye, resulting in vision loss in both eyes.

Have there been any trials of Bezafibrate?

Bezafibrate has been trialed for a number of conditions (other than mitochondrial disease) and is already licensed as a treatment. Because of this, the side effects are well known. There has been a clinical trial to study the effect of Bezafibrate in patients with mitochondrial disease. This was an open-label, non-randomised trial, meaning that both the researchers and the participants knew what treatment they were taking. The study aimed to assess the safety and tolerability of Bezafibrate in 10 patients with mitochondrial disease and determine its effect on mitochondrial function over 12 weeks.

What is the latest on this trial?

The results of this study were published in a medical journal in February 2020. The publication reported that six participants completed the trial with no clinically significant adverse events. In addition, an improvement in heart function was reported and there were fewer muscle fibres that showed problems with the energy chain when muscle samples were tested in the lab. However, this was accompanied by an increase in biomarkers of mitochondrial disease in blood samples and other signs of compromised energy production by the mitochondria. Therefore, it was concluded that although Bezafibrate could be potentially beneficial in the short term, concerns remained around the long-term health implications associated with its use. The researchers concluded that the evidence is not strong enough at present to recommend Bezafibrate as a treatment for mitochondrial disease caused by the m.3243A>G mutation.

What next?

A trial to study the efficiency of Bezafibrate (also known as Befizal) in 14 adult patients with LHON is still ongoing. In this trial, Befazil will be tested for 1 year to determine if it can improve vision. The study is due to be completed in 2022.

Is there more information?

The medical paper reporting the effects of Bezafibrate in mitochondrial disease can be found here.

More information on the trial of Befazil in LHON patients can be found here.

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