A VITAMIN-DERIVED MOLECULE REACHED ITS TARGET IN MITOCHONDRIAL DISEASE PATIENTS.

A Cambridge-led study gave nicotinamide riboside to eight people with mitochondrial DNA disease. It was well tolerated and showed early signs of working.

A VITAMIN-DERIVED MOLECULE REACHED ITS TARGET IN MITOCHONDRIAL DISEASE PATIENTS.

A small experimental study published in Genome Medicine reports the first human evidence that nicotinamide riboside can act on mitochondrial DNA disease.

WHAT HAPPENED

Eight people with heteroplasmic mtDNA disorders took 1,250-2,000 mg/day of nicotinamide riboside for four weeks. The molecule was bioavailable and well tolerated, and overall improved the timed up-and-go test. Muscle biopsies showed molecular changes in several participants.

WHY THIS IS HOPESLOP

These disorders affect roughly 1 in 8,000 people and have no treatment aimed at the underlying mechanism. A cheap, tolerable molecule reaching its target is a small, real door opening.

ORIGINAL SOURCEGenome Medicine / University of CambridgeRead the original report

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