Title : Evaluation of short term treatment with NAD+ precursor Nicotinamide Riboside on cognitive and mitochondrial function in patients with MELAS
Abstract:
Primary goal of this study was to measure effectiveness of nicotinamide riboside as direct precursor for coenzyme NAD+ in patients with mitochondrial DNA mutation 3443 and MELAS (Mitochondrial Encephalopathy, lactic acidosis and stroke like episodes) syndrome. Primary outcome were improved exercise tolerance (motor performance), decreased long term disability and improved cognitive performance. Oxidative stress, mitochondrial biogenesis and to Determine bioavailability after oral application. Selection of outcome measures for the future clinical included primary outcomes: exercise tolerance, disability rate and cognitive performance .Oxidative stress, lactate, ATP and sirtuin levels were measured as secondary outcomes. This pilot project succeeded to show improvement in endurance, exercise tolerance and cognitive performance and lower disability scores in patients with MELAS and there is a strong correlation with levels of biomarkers including oxidative stress, lactate ATP levels, nicotinamide and NAD+ levels and levels of sirtuin 1 without serious and intolerable side effects. This may offer very promising future treatment in patients with MELAS and 3443 mutation but also for patients with other mitochondrial diseases and cognitive dysfunction such as mild cognitive impairment and early dementia.