New Research: Statins May Show Promise as a Treatment for Dry Age-Related Macular Degeneration

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One of the most significant challenges facing eye and vision researchers is developing an effective treatment for dry age-related macular degeneration (AMD). Although there are now a number of well-regarded FDA-approved drug treatments for wet AMD, the key to effective dry AMD treatment remains elusive, although several potential treatments have emerged in recent years.

Current treatments for dry AMD include a number of non-drug-related measures, including (a) nutritional supplements recommended by the Age-Related Eye Disease Study 2 (AREDS2), and (b) controlling a range of lifestyle factors, including diet, weight, blood pressure, smoking, and blue and ultraviolet light exposure.

This month, researchers from Harvard Medical School and the University of Crete in Greece have published findings from a small phase 1/2 clinical trial in which 10 out of 23 subjects with dry AMD who were given high doses of the cholesterol-lowering drug Lipitor showed regression of drusen deposits (a buildup of waste materials beneath the retina, explained below) and demonstrated some improvements in visual acuity.

Although this research was conducted with a small number of subjects and is in its earliest stages, the results show promise as a potential treatment for dry AMD.

What are Statins?

Statins are a class of drugs, such as Lipitor, Crestor, and Zocor, that are used in the prevention and treatment of heart disease. They act to reduce the levels of fats, including triglycerides and cholesterol, in the blood by altering the enzyme activity in the liver that produces these fats. [Please note: Although millions of people take statins to control blood cholesterol levels, they do carry the risk of a number of side effects and require close medical supervision and monitoring.]

Statins and Dry Age-Related Macular Degeneration (AMD)

The research, entitled Regression of Some High-risk Features of Age-related Macular Degeneration (AMD) in Patients Receiving Intensive Statin Treatment, has been published online in the February 9, 2016 edition of EBioMedicine, an online-only, open-access journal, newly launched by the leadership of the journals Cell and The Lancet.

The authors are Demetrios G. Vavvas; Anthony B. Daniels; Zoi G. Kapsala; Jeremy W. Goldfarb; Emmanuel Ganotakis; John I. Loewenstein; Lucy H. Young; Evangelos S. Gragoudas; Dean Eliott; Ivana K. Kim; Miltiadis K. Tsilimbaris; and Joan W. Miller, who represent the following institutions: Massachusetts Eye and Ear Infirmary/Harvard Medical School, Boston, MA and the University of Crete, Heraklion, Crete, Greece.

About Dry Macular Degeneration

The dry (also called atrophic) type of AMD affects approximately 80-90% of individuals with AMD. Its cause is unknown, it tends to progress more slowly than the wet type, and there is not – as of yet – an approved treatment or cure. "Atrophy" refers to the degeneration of cells in a portion of the body; in this case, the cell degeneration occurs in the retina.

In dry age-related macular degeneration, small white or yellowish deposits, called drusen, form on the retina, in the macula – the small sensitive area in the center of the retina that provides clear central vision – causing it to deteriorate or degenerate over time.

Photograph of a retina with drusen

A retina with drusen

Drusen are the hallmark of dry AMD. These small yellow deposits beneath the retina are a buildup of waste materials, composed of cholesterol, protein, and fats. Typically, when drusen first form, they do not cause vision loss. However, they are a risk factor for progressing to vision loss.

More about the Research

Excerpted from High-Dose Statins May Ease Macular Degeneration for Some, via

High doses of cholesterol-lowering statin drugs – medicines such as Lipitor, Crestor and Zocor – may help people with … macular degeneration, a small study suggests. In the early-stage clinical trial, a team from Harvard Medical School assessed the effects of statin treatment in people with the dry form of age-related macular degeneration (AMD).

In the study, 23 patients with dry-form AMD were given a high dose (80 milligrams) of atorvastatin (Lipitor). In 10 of the patients, the fat deposits under the retina disappeared and they had a slight improvement in vision clarity, according to the study. It typically took a year to 18 months of treatment for these positive results to arise, the researchers reported. They noted that prior attempts to find ways to eliminate the fat deposits under the retina have failed.

However, "we found that intensive doses of statins carry the potential for clearing up the lipid [fat] debris that can lead to vision impairment in a subset of patients with macular degeneration," said study co-author Dr. Joan Miller. She is chair of ophthalmology at Harvard Medical School and chief of ophthalmology at Massachusetts Eye and Ear Infirmary and Massachusetts General Hospital, both in Boston. "We hope that this promising preliminary clinical trial will be the foundation for an effective treatment for millions of patients afflicted with AMD."

About the Study from EBioMedicine

From Regression of Some High-risk Features of Age-related Macular Degeneration (AMD) in Patients Receiving Intensive Statin Treatment:

There is a lack of effective therapies for dry age-related macular degeneration (AMD), one of the leading causes of blindness affecting millions. Although AMD shares similarities with atherosclerosis, prior studies on statins and AMD have failed to show improvement. A limitation of these studies has been the heterogeneity [i.e., diversity] of AMD disease and the lack of standardization in statin dosage.

Here, we present for the first time evidence that treatment with high-dose atorvastatin [Lipitor] (80 mg) is associated with regression of lipid [fat] deposits and improvement in visual acuity, without atrophy [i.e., degeneration of cells] or neovascularization [i.e., new blood vessels developing in the retina] in high-risk AMD patients.

Objective: We were interested in studying the effects of high-dose statins, similar to those showing regression of atherosclerotic plaques [i.e., a buildup of fats, cholesterol and other substances in and on the artery walls] in AMD.

Design: Pilot multicenter open-label prospective clinical study of 26 patients with diagnosis of AMD and the presence of many large, soft drusenoid [i.e., drusen, defined above] deposits. Patients received 80 mg of atorvastatin (Lipitor) daily and were monitored at baseline and every three months with a complete ophthalmologic exam, best corrected visual acuity, fundus [i.e., retinal] photographs, blood work, and optical coherence tomography [i.e., medical imaging technology that produces high-resolution cross-sectional and three-dimensional images of the eye].

Results:Twenty-three subjects completed a minimum follow-up of 12 months. High-dose atorvastatin resulted in regression of drusen deposits associated with vision gain (+3.3 letters) in 10 patients. No subjects progressed to advanced neovascular AMD.

Conclusions: High-dose statins may result in resolution of drusenoid pigment epithelial detachments (PEDs) and improvement in visual acuity, without atrophy or neovascularization in a high-risk subgroup of AMD patients. Confirmation from larger studies is warranted.

VisionAware will continue to report the results of this macular degeneration research as they become available.

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