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Could Statins Treat Dry Age-Related Macular Degeneration?

Research Findings

While more research is needed, a small clinical trial at Mass. Eye and Ear showed that some patients with dry age-related macular degeneration (AMD) improved when taking high doses of statins, a common drug to lower cholesterol.   

About two years ago, Mass. Eye and Ear physicians published results from a small clinical trial. The study suggested that some patients with dry age-related macular degeneration (AMD) may benefit from taking statins, a cholesterol-lowering medication, prescribed at high doses.

The physicians had prescribed 80mg of atorvastatin, the generic for Lipitor, to 23 patients with the dry form of the disease. But these patients also had some unique features. Their “drusen,” or the deposits of debris in the outer retina, was soft, fatty and lipid-like.

The results of this small study showed some potential: Of the 23 patients, 8 saw the deposits under their retinas disappear and 2 saw a reduction of the deposits — and vision tests in these patients showed mild improvements in visual acuity.

“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 and in patients with an accumulation of soft, lipid material,” said Demetrios Vavvas, M.D., Ph.D., a retina surgeon at Mass. Eye and Ear and first author on the study.

“We’ve found that intensive doses of statins carry the potential for clearing up the lipid debris that can lead to visual impairment in a subset of patients with macular degeneration,” added Joan W. Miller, M.D., senior author on the study, Chief of Ophthalmology at Mass. Eye and Ear and Mass General and Chair of Ophthalmology at Harvard Medical School.

A Leading Cause of Blindness

Affecting more than 150 million patients worldwide, age-related macular degeneration (AMD) is associated with an accumulation of drusen under the retina. Patients with AMD experience blurry vision or blindness in the center of the visual field.

The disease has two forms, “wet” and “dry,” with the dry form being more common, accounting for approximately 85 percent of cases. While ophthalmologists can slow vision loss from the wet form of the disease (using therapies developed in the early 2000’s at Mass. Eye and Ear), effective therapies for the dry form of the disease have long been lacking.

AMD and Atherosclerosis — a Connection?

Eye doctors and vision researchers have long suspected that there may be a connection between dry AMD and atherosclerosis, or a build-up of cholesterol in the arteries. In dry AMD, physicians often see soft, lipid-rich drusen in the outer retina, similar to the build-up of lipid material in the inner walls of blood vessels in atherosclerosis.

Statin use is widespread in middle-aged and older individuals, who also have an increased risk of AMD; however, previous studies have shown very little correlation between regular statin use and improvements in AMD. Before beginning the clinical trial, the study authors hypothesized that, due to the high level of variation in clinical presentation of the disease, patients with soft, lipid-rich drusen may respond better to statins prescribed at higher doses.

Should You Take Statins for Dry AMD?

For now, the best advice is to follow the recommendations of your eye care provider. However, the study authors hope to have a more definitive answer soon.

The great potential of this discovery led to the launch of Drusolv Therapeutics, a Mass. Eye and Ear spin-out company, to develop and commercialize a statin medication specifically tailored for the treatment of AMD.

Clinical trials are expected to begin later this year. For more information, please contact OphthalmologyClinicalResearch@meei.harvard.edu.

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