Mito research around the world


What is Acipimox?

Acipimox is a drug that is currently used to treat high cholesterol and can help control diabetes. In addition to this, preclinical research studies have suggested that Acipimox can improve mitochondrial function within muscle cells.

How does it work?

Acipimox is thought to increase levels of an enzyme called NAD+ (nicotinamide adenine dinucleotide). NAD+ has several roles in the cell and is important for energy production.

What is it supposed to do?

Acipimox is thought to boost energy production in the mitochondria found within muscle. This could reduce muscle weakness in patients with mitochondrial disease.

Is it a cure?

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

What types of Mitochondrial Disease could it treat?

Acipimox is due to be trialled in patients with mitochondrial disease and evidence of muscle weakness (myopathy). In the first instance, this will involve adult patients with a particular type of genetic error causing mitochondrial disease (the m.3243A>G mutation or single mitochondrial DNA deletion).

Have there been any trials of Acipimox?

Acipimox 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 and effective dosage of Acipimox are well known. This kind of ‘drug repurposing’ is beneficial and could provide a faster and much cheaper way of getting drugs to patients.

MRC funding was recently awarded for a clinical trial in the UK to study the effect of Acipimox in patients with mitochondrial disease. This will be a randomised, double blind, placebo-controlled trial that aims to assess the efficacy of Acipimox in patients with mitochondrial disease.

What is the latest on this trial?

The trial is currently looking to recruit up to 120 adult patients who meet specific enrolment requirements. This recruitment will initially be done through the MRC MitoCohort.

What next?

If the results of this study show clinical benefits for adult patients, the drug could be developed for children with mitochondrial disease and muscle weakness.

How do I get more information on this trial?

For additional information, or to be considered for the trial, please contact [email protected] .

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