A recent study has found that Niagen Bioscience's nicotinamide adenine dinucleotide (NAD+) precursor, Niagen, can benefit patients with Werner syndrome (WS).
Notably, the nicotinamide riboside-based nutraceutical was found to both elevate circulating NAD+ levels significantly and improve clinical markers of cardiovascular and skin health in patients with WS.
Published in Aging Cell, these findings uncover the potential of Niagen as a treatment for the rare genetic disorder, which is characterised by the rapid ageing and premature mortality of those who suffer with it.
Tackling WS with Niagen
Werner syndrome is a rare genetic disease that impacts between 1:380,000 to 1:1m people globally, though its prevalence is much higher in Japan, with around 1 in 100,000 having the condition.
It is commonly characterised by rapid ageing, causing many skin-related issues similar to scleroderma, as well as ischemic heart disease.
There is currently no cure for the condition, though there are a range of treatments available for when symptoms arise.
To assess the potential of Niagen to reduce symptoms of WS, researchers at the Chiba University Hospital, Japan ran a double-blind, placebo-controlled study involving 12 individuals with the condition.
During the study period, participants were either given 1000mg of Niagen or a placebo daily for 26 weeks, after which they were switched to the opposite treatment for an additional 26 weeks.
Interestingly, supplementation with Niagen increased blood NAD+ levels by around 140%, while filtration by the kidneys increased after NR supplementation.
Niagen also reduced arterial stiffness and the number of large high-density lipoprotein (HDL) particles, suggesting the ingredient could offer cardiovascular benefits.
Meanwhile, Niagen could also reduce skin ulcer size and heel pad thinning, inferring that the product could be used to treat the skin-related issues associated with WS.
Although mild adverse events were reported in the population randomised to Niagen, there were less adverse events in this group than the placebo.
This study builds on the growing body of clinical research demonstrating Niagen’s potential in rare, age-related diseases, with the ingredient also showing promise for the treatment of Ataxia Telangiectasia.
"We hope that the results of this study and our current work in rare disease will accelerate research into premature ageing disorders and common age-related diseases — ultimately helping to extend healthspan and improve quality of life," noted Dr. Koshizaka, lead author of the study.
The FDA previously granted NR Orphan Drug Designation (ODD) and Rare Paediatric Disease (RPD) designation for the treatment of AT.
"This study marks the first clinical trial using Niagen in those with Werner syndrome, and the findings—particularly improvements in cardiovascular markers—are promising," added Vilhelm Bohr, Affiliate Professor in Genome Instability and Neurodegeneration at the University of Copenhagen and Scientific Advisor to Niagen Bioscience.
"As Werner syndrome is a well-established model for normal ageing, these results also suggest that NAD+ supplementation may support healthier ageing in the broader population.”
This study represents the first clinical evaluation of Niagen in WS and supports further investigation of NAD+ augmentation as a therapeutic strategy in rare progeroid diseases.