A common cholesterol-lowering drug can restore vision to patients with a hard-to-treat version of the leading cause of blindness in the developed world, research has shown.
High-dose treatment with the statin Lipitor cleared away fatty deposits behind the retina, leading to visual improvement in 10 patients with age-related macular degeneration (AMD).
Scientists hope future larger trials will show that the drug has the potential to halt progression and even reverse the disease in some cases.
US lead researcher Professor Joan Miller, chair of ophthalmology at Harvard Medical School, said: "We found that intensive doses of statins carry the potential for clearing up the lipid debris that can lead to vision impairment in a subset of patients with macular degeneration.
"We hope that this promising preliminary clinical trial will be the foundation for an effective treatment for millions of patients afflicted with AMD."
AMD, which affects more than 150 million people worldwide, is a progressive disease caused by the accumulation of fatty lipids and protein under the retina, the "camera" at the back of the eye.
Over time, patients experience increasingly blurred vision or blindness emerging from the centre of the visual field.
There are two forms of AMD, known as "wet" and "dry". Of the two, the more common "dry" form that accounts for 85% of cases is more difficult to treat, and lacks effective therapies.
Experts have suspected there may be a link between dry AMD and atherosclerosis, the narrowing of arteries by fatty deposits on blood vessel walls that can lead to heart attacks and strokes.
Cholesterol-lowering statins are taken by millions of middle-aged and older people to reduce the risk of atherosclerosis.
Previous studies have shown little correlation between regular statin use and AMD improvement.
But Prof Miller's team suspected a specific sub-group of patients may benefit from higher doses of the drugs than those typically prescribed.
Dr Demetrios Vavvas, from Massachusetts Eye and Ear Infirmary in the US, who co-led the trial, said: "Not all cases of dry AMD are exactly the same, and our findings suggest that if statins are going to help, they will be most effective when prescribed at high dosages in patients with an accumulation of soft lipid material.
"These data suggest that it may be possible to eventually have a treatment that not only arrests the disease but also reverses its damage and improves the visual acuity in some patients."
In total 23 patients were recruited who had dry AMD marked by soft lipid deposits. They were prescribed a high 80 milligram daily dose of atorvastatin, which is marketed under the brand name Lipitor. Several generic versions of the drug were also used.
Of the group, 10 patients experienced an elimination of the deposits under their retinas and a mild improvement in visual acuity.
Other techniques that have attempted to remove lipid deposits from patients with dry AMD have mostly failed and allowed the disease to progress.
The scientists, whose findings appear in the new online journal EBioMedicine, now plan to extend their research to a larger multi-centre trial with many more patients.
Dr Vavvas added: "This is a very accessible... approved drug that we have tremendous experience with.
"Millions of patients take it for high cholesterol and heart disease, and based on our early results, we believe it offers the potential to halt progression of this disease, but possibly even to restore function in some patients with dry AMD."