New drug could prevent blindness caused by inherited eye disease

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A new drug which could help prevent sight loss caused by a currently untreatable condition is being trialled for the first time in the UK.

Stargardt’s disease, also known as Stargardt’s 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.

There is currently no available treatment, with patients advised to avoid exposure to bright light and wear sunglasses to try to slow progression of the disease.

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

The medication, which is taken orally, removes the lipid – or fat – deposits, known as lipofuscin, which build up in the back of the eye.

Prof Lotery said: “This disease is one of the most common forms of inherited retinal disease and results in progressive sight loss through adolescence and into adulthood.

“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, specialist in inherited retinal diseases at Radboud University Medical Centre in the Netherlands, said: “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, while Prof Lotery’s work in Southampton is supported by the charity Gift of Sight which he established in 2004 to raise money for research into the treatment of complex eye disease.

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