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Lily Research News


3 October 2017

Supporting medical research into mitochondrial disease is a key part of our work at The Lily Foundation. It's consistent with our long-term goal of finding a cure for mito, and also brings life-changing benefits to people who are living with the disease today. Here we look at a promising study that’s currently underway at University College London.

A hand in a white medical glove hold a testtube full of red liquid

What is Nicotinamide Riboside? Nicotinamide Riboside (NR) is a form of vitamin B3 that has been shown to increase the number of healthy mitochondria in animals with mitochondrial disease. Researchers at University College London and Cambridge University are now working towards testing NR to confirm its effects in human patients.

How might NR benefit patients? Using NR to increase the number of healthy mitochondria in cells could help to reduce the symptoms of mitochondrial disease. Mitochondrial diseases are among the most common genetic disorders affecting 1 in 4,300 people, but there are currently no effective treatments to halt or reverse their progression.

What are the challenges? One obstacle that could potentially limit NR's transition to human trials relates to how much of the vitamin is absorbed into the bloodstream. Researchers have found that in standard tablet form the quantity of NR that enters the bloodstream is potentially insufficient for an effective treatment.

What solutions are the team working on? Proposals have been made to develop a modified release version of NR to improve uptake and sustain levels in the blood. Plans are also in place to create an intravenous (injectable) form of NR that can be used in the event that a patient is unable to swallow tablets (a common scenario when people with mitochondrial disease become acutely unwell).

What's the word from the lab? Dr Rob Pitceathly, who heads up the research at UCL, said: "The grant awarded by the Lily Foundation will enable development of both a modified release tablet and injectable form of Nicotinamide Riboside. This work will maximise the potential clinical benefits conferred by NR and is a prelude to using it in a human clinical trial."

You can find out more about all the projects currently funded by The Lily Foundation.

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