Mito research around the world

An exciting time for mitochondrial research

This is a very exciting time for research into mitochondrial disease. Growing awareness about the condition and improved diagnosis have resulted in more global research centres undertaking valuable work on potential drug treatments.

Scientists have linked mitochondrial dysfunction with other serious diseases including cancer, diabetes, Alzheimer's and stroke, making this an even more vital field of study.

While there have been several exciting breakthroughs in recent years, many studies are still a long way from clinical trials. The information provided here focuses on the most promising looking treatments, which we hope will be of benefit to patients in the near future.

Current research projects around the world:

Bezafibrate

Bezafibrate is a drug commonly used to lower the levels of fats in the blood, such as cholesterol. Research suggests that Bezafibrate can improve mitochondrial function within cells, which could potentially reduce mitochondrial disease symptoms in patients.

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Acipimox

Research suggests that Acipimox, a drug used to treat high cholesterol and can help control diabetes, could be used to improve mitochondrial function and boost energy production in muscle cells.

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MT1621 (nucleoside therapy)

MT1621, also known as nucleoside therapy or deoxynucleoside ‘substrate enhancement therapy’ (SET), is a potential treatment for a mitochondrial DNA depletion syndrome called TK2 deficiency (TK2d).

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Cysteamine Bitartrate (RP103)

Clinical trial discontinued.

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EPI-743

In clinical trial.

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Elamipretide (Bendavia, MTP-131)

Clinical trial discontinued.

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Genome Editing using CRISPR/Cas

In development.

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GS010 Gene Therapy

In clinical trial.

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Idebenone (Raxone)

In clinical trial.

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JOTROL

Clinical trial planned.

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Sonlicromanol (KH-176)

In clinical trial.

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KL-1333

In clinical trial.

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NV354

Pre-clinical research.

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Nucleoside Bypass Therapy & Mitochondrial DNA Depletion Syndromes

Pre-clinical research.

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Omaveloxone

In clinical trial.

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