Major NIHR grant awarded to trial a form of vitamin B3 as a treatment for glaucoma

The trial will eventually recruit 496 glaucoma patients across seven UK sites, with a pilot study starting at Moorfields and Kings College Hospital in London along with a third site yet to be confirmed. It will evaluate whether NAM, a form of Vitamin B3, which replenishes molecules important for the functioning of mitochondria (the powerhouses of cells), protects glaucoma patients from progressive vision loss.

The trial builds on collaborative research at Moorfields, UCL IoO and the UCL Queen Square Institute of Neurology (IoN) showing that patients with both high- and low-tension glaucoma have lower mitochondrial function than healthy patients; and also that patients with ocular hypertension (higher than normal pressure inside the eye), but no signs of progression to glaucoma, have higher mitochondrial function.

The study will measure the impact of NAM on the capacity of mitochondria in peripheral blood lymphocytes (freely circulating white blood cells) to produce adenosine triphosphate (ATP), which provides energy to help cells function. The study will also investigate biomarkers (biological signs) that can predict disease progression for glaucoma.

Starting in August 2022, the trial will recruit a diverse group of patients recently diagnosed with glaucoma at NHS sites around the country. If treatment with NAM proves successful, it could significantly reduce the damage to vision caused by glaucoma and the cost of treatment for the NHS.

The trial has been funded by the National Institute for Health Research (NIHR) Efficacy and Mechanism Evaluation (EME) Programme. The programme funds ambitious studies that evaluate interventions that have the potential to make a significant change in the promotion of health, treatment of disease and improvement of rehabilitation or long-term care. Within these studies, EME supports research in the mechanisms of diseases and treatments. EME is a partnership between the Medical Research Council and the NIHR.

Prof. Ted Garway-Heath, lead applicant for the grant, said: “We hope to find a treatment that isn’t directed at pressure in the eye, but that addresses the susceptibility of the patient to glaucoma. We also hope to identify a blood test to identify which patients will benefit from the new treatment.”

The new study will be based primarily at UCL IoO, which has recently acquired a Seahorse Analyzer (with funding from Moorfields Eye Charity), a machine that allows scientists to look at mitochondrial function within cells.

Leading the clinical trial alongside Prof. Garway-Heath is Gerassimos Lascaratos, consultant eye surgeon at King’s College Hospital. The other principle investigators are James Kirwan at Portsmouth Hospitals, David Broadway at Norfolk and Norwich University Hospitals, Rupert Bourne at the Cambridge Eye Research Centre (Cambridge University Hospitals), Anthony King at Nottingham University Hospitals and Augusto Azuara-Blanco at Queen’s University Belfast.