Ophthalmologists at the University Hospital Southampton NHS Trust will be the first in the UK to trial the drug remofuscin. Researchers across Europe are hopeful remofuscin could be the key to treating the devastating eye disorder known as Stargardt's disease.
Stargardt's disease is an inherited disease that causes damage to a patient's retinae. Common symptoms of Stargardt's disease include issues with central vision and blurred vision and are most often noticed in childhood. There are a few rare cases of Stargardt's disease that don't present symptoms until adulthood.
Scientists involved in developing remofuscin say this drug is different from any other therapy trialed before. They point out that remofuscin has effectively destroyed lipofuscin in pre-clinical trials. Lipofuscin is a fatty, yellow compound that gets stuck in Stargardt's disease patient's eyes.
Most doctors believe Stargardt's disease is primarily caused by a mutation in the ABCA4 gene. When people don't have this ABCA4 gene, they can't eliminate byproducts of vitamin A as effectively as a normal eye, which leads to a buildup of lipofuscin.
This remofuscin trial is expected to cost a total of £5 million. The European Commission's H2020 Horizon program will fund the majority of this research.
Carel Hoyng is the head researcher on this remofuscin clinical study. Dr. Hoyng works at the Radboud University Medical Center in the Dutch city Nijmegen.
In addition to the UK and the Netherlands, researchers in Italy, Germany, and Norway are actively involved in this study.
The EU's Horizon 2020 is a major research campaign that began in 2014 and will last until 2020. Over the course of these seven years, the EU will donate €80 billion to help fund clinical trials.
Approximately one in every 9,000 people have Stargardt's disease. Stargardt's disease patients are at a higher risk of developing age-related macular degeneration (AMD) and many become visually handicapped by their 20s.
As of today, there's no official cure for Stargardt's disease. There are, however, many electronic devices that can help manage their symptoms. Ophthalmologists strongly recommend Stargardt's disease patients avoid smoking, always wear sunglasses, and only take vitamin A supplements with a doctor's approval.