Nicotinamide Riboside (NR), a precursor of nicotinamide adenine dinucleotide (NAD+), may be useful in the treatment of symptoms of Ataxia Telangiectasia (AT), according to a prospective, single-arm study published in Movement Disorders.
 
AT, also known as Louis-Bar syndrome, is a rare neurological disorder which affects the part of the brain that controls motor movement and speech, stemming from a DNA repair deficiency. It is characterized by premature aging, cerebellar degeneration, immunodeficiency, and cancer predisposition. Individuals diagnosed with AT frequently experience profound challenges in motor function, characterized by neurodegenerative conditions like cerebellar ataxia, resulting in impaired movement and coordination and they may exhibit oculomotor apraxia, difficulties controlling eye movements. These factors often result in a loss of independent walking ability, typically necessitating the use of a wheelchair by the age of ten.
 
The present study was conducted by a team of researchers at the University of Oslo Arkershus University Hospital in Norway, and was supported by ChromaDex, which provided researchers with its branded NR ingredient Niagen, through the company’s external research program.
 
In total, ten participants completed the study, which involved treatment with NR over a period of two years, during which time they took part in neuromotor test panels.
 
By the 18-month time point, all participants saw significant improvements in test panel scores, which was primarily driven by improvements in the sub scores of coordination and eye movement.
 
A comparison to historical data revealed that the progression of certain neuromotor symptoms was slower than anticipated over the two-year time frame.
 
This was the second published study analyzing the therapeutic effects of NR supplementation in AT patients, and was the longest Niagen NR trial to date with results providing evidence that long-term supplementation is safe and well-tolerated. In the first study, which spanned four months, daily NR treatment was linked to improvements in ataxia scores across 24 patients, which worsened after NR withdrawal. In immunodeficient patients, the mean serum immunoglobulin-G concentration increased substantially until the end of the study period.
 
“While the exact mechanisms underlying cerebellar degeneration and neurodegeneration in AT are not fully understood, research indicates inability to repair and respond appropriately to DNA damage,” said Hilde Nilsen, PhD, lead author of the study. “AT research has shown that DNA damage depletes NAD+, the crucial cellular coenzyme that plays an essential role in mitochondrial function, DNA repair, and cellular energy production. This growing body of research is promising as it demonstrates that NR supplementation may be a potential therapeutic strategy for AT patients.”