Long-Term Nicotinamide Riboside Use Improves Coordination and Eye Movements in Ataxia Telangiectasia

Area of Science:

• Neurology
• Biochemistry
• Clinical Trials

Background:

• Nicotinamide riboside (NR) supplementation shows promise in ameliorating neuropathology in animal models of ataxia telangiectasia (A-T).
• Previous short-term human studies indicated neurological benefits of NR in A-T patients.

Purpose of the Study:

• To evaluate the safety and efficacy of long-term NR supplementation in individuals diagnosed with A-T.
• To assess the impact of 2-year NR supplementation on neurological outcomes and safety parameters in A-T patients.

Main Methods:

• A 2-year, single-arm, open-label clinical trial involving individuals with A-T.
• Safety was monitored through biomarkers and clinical examinations.
• Neuromotor function was assessed using standardized tests, with results compared to historical disease progression data.

Main Results:

• NR supplementation rapidly increased NAD+ concentrations in peripheral blood, stabilizing at elevated levels.
• The treatment was generally well-tolerated by participants.
• Significant improvements in neuromotor test scores, particularly coordination and eye movements, were observed in most participants, indicating slower than anticipated disease progression.

Conclusions:

• Long-term NR supplementation is safe and well-tolerated in patients with A-T.
• NR treatment demonstrates efficacy in improving motor coordination and eye movements across all age groups of A-T patients.
• These findings support NR as a potential therapeutic agent for managing neurological symptoms in A-T.