Werner syndrome (WS) is a rare genetic disorder characterized by accelerated aging, affecting approximately nine individuals per million in Japan. Patients typically begin experiencing symptoms such as gray hair, cataracts, diabetes, and severe skin ulcers by their twenties, leading to significant health challenges and early mortality due to cardiovascular diseases or cancer. Currently, effective treatment options for WS are limited.
A recent study conducted by a team led by Associate Professor Masaya Koshizaka at Chiba University explored the effects of nicotinamide riboside (NR), a precursor to the biomolecule nicotinamide adenine dinucleotide (NAD+), in WS patients. The study, published in Aging Cell in June 2025, marks the first rigorous clinical trial assessing NR's safety and effectiveness in this population.
The randomized, double-blind, placebo-controlled trial involved enrolling WS patients to receive either NR or a placebo over a 26-week period, followed by a crossover to the alternative treatment. Key metrics evaluated included NAD+ blood levels, skin ulcer size, arterial stiffness, and kidney function.
Results indicated that NR supplementation significantly increased NAD+ levels and improved arterial stiffness, reduced the area of skin ulcers, and appeared to slow kidney dysfunction progression. Notably, these benefits were observed without serious side effects. The research suggests that restoring NAD+ levels may address fundamental mechanisms in WS, highlighting NR's potential as a therapeutic option.
While further studies are necessary to confirm these findings, this research provides new insights into treatment possibilities for WS and may inform broader applications in addressing age-related health issues. Dr. Koshizaka expressed hope that this work could enhance understanding of both WS and other premature aging disorders, ultimately improving health outcomes for affected individuals.