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LifeArc Centre to Treat Mitochondrial Disorders

The LifeArc Centre for Rare Mitochondrial Diseases (LAC-TreatMito), led by Professor Patrick Chinnery at the University of Cambridge, aims to improve diagnostics and develop treatments for mitochondrial diseases.
Details
Principal Investigator
Professor Patrick Chinnery
Institute
University of Cambridge
Duration
Five years
Total cost
£1,142,945
Conditions
Mitochondrial diseases
Year
2024

Background

Mitochondrial diseases are a group of conditions in which small cellular structures called mitochondria don’t work properly. Mitochondria are the main producers of energy in cells and often referred to as the ‘powerhouse’ of the cell. Mitochondrial diseases mainly affect parts of the body that require the most energy to work properly, such as muscles, the heart, liver, brain, nerves and pancreas.

There are currently no cures for these conditions, and the development of treatments is often slowed by challenges in diagnosis and a lack of available tests, also known as biomarkers, to measure how mitochondria work in those people with symptoms of mitochondrial condition

The centre led by Professor Patrick Chinnery at the University of Cambridge will work to address this hold up in getting treatments from laboratory to clinic by setting up a national platform to bring new treatment approaches into clinical trials.

With Professor Chinnery as the Principal investigator, the centre boasts some of the UK’s leading experts in the field of mitochondrial disease, including Professor Rita Horvath, University of Cambridge, Professor Robert Taylor, Newcastle University, Professors Robert Pitceathly and Mike Hanna, UCL. Clare Chatfield from the Lily Foundation will also be part of the Centre, and it will have formal links across the UK, including with teams in Manchester, Birmingham and Oxford.

What are the aims of the project?

The team aims to show within five years that six new advanced therapies (therapies that are based on genes, tissues or cells, such as gene therapies, exon-skipping, cell therapies and others) have the potential to work. They will also work to develop new biomarkers for mitochondrial diseases in clinical trials.

This national platform will improve the diagnosis of mitochondrial diseases nationwide, connecting patient groups, knowledge and infrastructure to continue bringing new treatments into trials even after this grant has finished. This will be achieved in partnership with the Lily Foundation, linking patients with universities and industry globally.

As part of this grant award, along with supporting the research itself, we will be supporting five post-doctoral medically qualified researchers. These are doctors who have got a PhD and want to develop their research career further. Our support will help these early career researchers to become independent researchers in the future who will form part of the next generation of neuromuscular specialists contributing to research beyond mitochondrial diseases and other muscle wasting conditions through the NHS.

The importance of this study

Improving the diagnosis of mitochondrial diseases and bringing new treatments into the clinic will have a beneficial effect on people with these conditions. Early diagnosis means treatment could be given sooner, preventing the early onset of symptoms and hopefully reducing the speed of progression. Importantly, this is a large, nationwide study, and any results could impact how the condition is managed across the UK.

Acknowledgements

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