Nicotinamide Riboside Extends Lifespan and Restores Cardiac Function in Friedreich’s Ataxia: Preclinical Findings

Synopsis

Friedreich’s ataxia (FRDA) is a rare inherited disease caused by reduced production of frataxin, a mitochondrial protein essential for iron-sulfur cluster formation. The condition leads to movement problems, heart abnormalities, and eventual heart failure. In this study, researchers used mice with reduced frataxin levels (shFxn mice), which developed motor deficits, early death, and heart enlargement resembling early heart changes seen in young FRDA patients. When treated with nicotinamide riboside (NR) or nicotinamide mononucleotide (NMN)—two NAD+ precursors that support mitochondrial energy metabolism—the mice lived longer, showed reduced heart hypertrophy, and had better control of heart function. Although most gene and metabolic disturbances caused by frataxin loss were not corrected, glutathione levels increased, suggesting improved antioxidant defense. These findings show that boosting NAD+ modestly protects the heart and delays disease progression in FRDA, supporting further research into NAD+-targeted therapies for mitochondrial and cardiac complications.

Journal

JCI Insight