Treatment trial for Stargardt disease reaches the UK

Scientist in lab

Researchers in the south of England will become the first in the UK to trial a new drug that could prevent sight loss caused by Stargardt disease, a currently untreatable condition.

Stargardt disease, also known as Stargardt macular dystrophy, affects the area of the retina called the macula and causes a reduction in central vision.

It is the most common form of juvenile macular degeneration and affects around one in 10,000 children who suffer a gradual decline in vision which leads to blindness in adulthood.

Professor Andrew Lotery, from University Hospital Southampton NHS Foundation Trust, is to lead the UK’s team in a £5 million study of the drug remofuscin with colleagues in the Netherlands, Italy, Germany and Norway.

Speaking about the new trial, Professor Lotery said: “There is no approved treatment available for Stargardt's, so we are very excited about the opportunity to test remofuscin and offer these patients the hope that we may be able to stop the progression of the condition.”

Professor Carel Hoyng, who is a specialist in inherited retinal diseases at Radboud University Medical Center in the Netherlands and is the study’s principal investigator, added: “This drug’s potential goes beyond existing approaches. So far the attempt was to keep lipofuscin levels the same, but remofuscin has been shown to actually remove existing lipofuscin in pre-clinical models and this may result in significant patient benefit.”

The study has received funding from the European Commission’s H2020 programme. Professor Lotery’s work in Southampton is supported by Gift of Sight, a charity that was established in 2004 to raise money for research into the treatment of complex eye disease.

The study has not yet been set up and is not currently recruiting.