High-dose statin treatment improves symptoms of macular degeneration

by Bruce Sylvester: Investigators from a phase l/II clinical trial report that high-dose atorvastatin treatment of patients with the dry form of age-related macular degeneration (AMD) resulted in a regression of lipid deposits and improvement in visual acuity, without progression to advanced disease.

Their findings were published in EBioMedicine on Feb. 4, 2016

“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,” said Joan W. Miller, M.D., the Henry Willard Williams Professor and Chair of Ophthalmology at Harvard Medical School and Chief of Ophthalmology at Massachusetts Eye and Ear 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,” she added.

As background, the researchers noted that in spite of the availability of effective anti-angiogenic therapies for the less prevalent neovascular form of AMD, there are a lack of effective treatments for the more prevalent dry form of AMD.

For this multicenter, open-label prospective study, the investigators enrolled 26 patients with diagnosis of AMD and the presence of many large, soft drusenoid deposits.

Subjects received 80 mg of atorvastatin daily, and they received a complete ophthalmologic examination at baseline and every 3 months.

Twenty-three subjects completed a minimum follow-up of 12 months.

In 10 subjects, high-dose atorvastatin therapy resulted in regression of drusen deposits (an elimination of the deposits under the retina which is associated with vision gain) and mild improvement in visual acuity (+3.3 letters, p = 0.06).

None of the subjects who completed the study had progressed to advanced neovascular AMD.

“Not all cases of dry AMD are the 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” said Demetrios Vavvas, M.D., Ph.D., a clinician scientist at Massachusetts Eye and Ear and Co-Director of the Ocular Regenerative Medicine Institute at Harvard Medical School. “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.”