UK government approves £1.6 million study into new mitochondrial disease treatment

13 December 2017

Scientists at the Wellcome Centre for Mitochondrial Research, Newcastle will test Acipimox, a drug currently used to treat high cholesterol in a range of conditions including heart disease and some types of diabetes. The drug has been shown to improve energy production in muscle cells, which could benefit patients with mitochondrial disease.

The project, which has been two years in development, has been awarded £1.6 million in funding by the Medical Research Council, the government body responsible for coordinating and funding medical research in the UK.

The clinical trials will involve patients with mitochondrial disease who have evidence of muscle weakness (myopathy). If clinical benefits are seen, the drug could then be developed to treat children with mitochondrial disease.

Dr. Grainne Gorman, who is leading research on the project, highlighted the advantages of working with a drug that has already been used to treat other conditions:

“The benefits are that we already understand the behaviour of the drug in humans. We know about the side effects, we know how to administer the drug, and we have some understanding about effective doses. This method potentially provides a faster and less expensive pathway to address the urgent need for drug treatments in mitochondrial diseases.”

The study is being hailed as an important step forward for research into mitochondrial disease, an incurable and severely life-limiting genetic condition that affects one baby born in the UK every day. Alison Maguire, Head of Research and Finance at The Lily Foundation, a UK charity that supports families affected by mitochondrial disease, commented:

“We are proud to have been involved in helping to secure this funding, and delighted that these important patient trials will take place in the UK. Dr. Gorman and her team have worked hard to involve patients in the design of the project from the very beginning. This kind of patient-researcher engagement is crucial to ensure that new drug research puts patient interests first, and we strongly believe this will maximise patient participation and ultimately improve the outcome of the trial."