Nicotinamide riboside shows promise in treating symptoms of Werner syndrome

Nicotinamide riboside shows promise in treating symptoms of Werner syndrome

Werner syndrome (WS) is a rare genetic disorder that causes premature aging and serious complications. In the first clinical study of its kind, researchers from Japan with Niaggen Bioscience examined the effects of nicotinamide riboside (NR), a vitamin B3 derivative, in patients with WS. NR supplementation significantly improved cardiovascular health, reduced skin ulcer areas, and slowed renal function decline, adding promising therapeutic potential for a disease that currently lacks effective treatment.

Werner syndrome (WS), a rare genetic disorder that causes accelerated aging. By their twenties, patients develop gray hair, hair loss, cataracts, diabetes and other age-related conditions usually seen in older people. In addition, patients develop severe and untreatable skin ulcers, often requiring limb amputation, and face early death from cardiovascular disease or cancer. There are no effective treatment options for this debilitating condition, which affects approximately nine per million people in Japan.

Interestingly, a recent study from the Bohr Lab (Nat Commun2019 November 21st; 10 (1): 5284) showed that patients with WS model systems and patients had reduced nicotinamide adenine dinucleotide (NAD+), a biomolecule crucial for cellular energy production, DNA repair and various metabolic processes. This finding suggested that NAD+ increase may contribute to disease progression. While direct NAD+ supplementation is not possible in mammals, using its precursor nicotinamide riboside (NR) from Niagen Bioscience has shown promising results in animal studies, extending lifespan and protecting against age-related decline. In human clinical trials, NR has also shown benefits against chronic inflammation, metabolic disorders, and muscle weakness in various populations. However, the effects of NR in WS have remained largely unexplored – until now.

In a recent study, a research team led by Associate Professor Masaya Koshizaka from the Center for Preventive Medical Sciences, Chiba University/Department of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Japan, conducted the world's first rigorous clinical trial of NR in patients with WS. Your newspaper, published inAging cellOn June 3, 2025, University President Koutaro Yokote, Assistant Professor Hisaya Kato, Associate Professor Yoshiro Maezawa and Assistant Professor Mayumi Shoji, all from the University of Chiba at the University of Chiba, and Assistant Professor Mayumi Bohr from the University of Copenhagen, Denarkern, Denmark, were co-authorized.

This groundbreaking work involved a randomized, double-blind, placebo-controlled trial to evaluate the safety and effectiveness of NR supplementation. The research team enrolled patients with WS in a crossover design in which participants received a daily dose of NR or a placebo for 26 weeks, followed by treatments for an additional 26 weeks. Researchers tracked NAD+ blood levels, skin ulcer size, arterial stiffness and kidney function.

NR supplementation significantly increased NAD+ levels in patient blood compared to placebo. Importantly, NR improved arterial stiffness (a marker of cardiovascular disease), reduced skin ulcer area, and appeared to slow the progression of kidney dysfunction - all without serious side effects. In addition, a comprehensive study of metabolites in the blood found that NR treatment reduced levels of creatinine and other compounds associated with kidney dysfunction. This suggests that NR may help protect renal function and address another serious complication of WS.

Dr. Yasmeen Nkrumah-Elie, global director of Niaggen Bioscience’s external research program called CERP, commented “This study represents a significant step forward in understanding how NAD+ restoration with NR can help address the underlying biology of WS. By supporting cardiovascular, skin and kidney health, NR shows the potential to improve the quality of life of patients with this devastating disease. We are proud to support Chiba University's groundbreaking research as part of our ongoing commitment to advancing NAD+ science for rare and underserved diseases.“

The multiple benefits of treatment in many different organ systems indicate that NAD+ increase may be a fundamental mechanism in WS that can be targeted therapeutically. “Our results suggest that NR could serve as a valuable treatment option for two major symptoms, atherosclerosis and skin ulcers, as well as for the prevention of renal function decline"Explains Dr. Koshizaka. The results are particularly significant because intractable skin ulcers affect well over 70% of patients with WS, often leading to amputation, while cardiovascular disease remains a major cause of early mortality in this population.

Although larger studies are needed to extend these results, this groundbreaking research offers new hope to patients with WS who have long lacked effective treatment options. Beyond its immediate impact on this rare disease, the study also provides valuable insights into the biology of aging and potential interventions to further address age-related decline.

“We hope that our work will accelerate studies not only on WS but also on other premature aging disorders and general age-related diseases.“Closes Dr. Koshizaka.

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