Stargardt pill continues to slow sight lossPosted: Tuesday 28 November 2023
A drug being trialled as a potential treatment for Stargardt disease has been shown to slow down the rate of sight loss.
A clinical trial where patients were given Remofuscin tablets over two years has been shown to slow down the rate of progression in the disease, which affects approximately one in 10,000 people.
The drug works by removing a material called lipofuscin, which builds up in the macula in Stargardt disease and damages the important retinal pigment epithelium (RPE) cells.
Efforts to remove lipofuscin from the RPE cells, either through natural processes or existing therapies, have proven unsuccessful but Remofuscin is looking promising as an effective treatment to preserve sight.
As well as Stargardt disease, Remofuscin is also being tested as a potential treatment for dry age-related macular degeneration (AMD), having completed Phase I trials alongside the Phase II study for Stargardt.
Professor Andrew Lotery of the University of Southampton Hospitals Trust, who worked closely on the Remofuscin project, said: “It’s been a successful trial to date and we’ve seen some promising findings in terms of reducing retinal thinning. Further studies to evaluate Remofuscin as a treatment for both Stargardt and dry AMD are now needed and that is being explored.”
The findings have been reported by biopharmaceutical company Katairo GmbH, which recruited 87 patients for the first stage of the trial. Sixty-one people completed the second year of treatment, of which two thirds of patients received two Remofuscin tablets daily. One third of participants received a placebo.
Welcoming, the latest results, managing director of Katairo GmbH, Dr Wolfgang Klein, said: “We are very pleased with the outcome of this study. Retinal thinning is known to precede the formation of atrophy in Stargardt disease. These results with Remofuscin are good news for patients hoping to finally have a treatment slowing the disease process.”
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Stargardt disease is a genetic condition caused by a tiny alteration in a single gene. It is the most common form of juvenile macular dystrophy.
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