Macular Degeneration May Improve with High Dose Statin Therapy

MedicalResearch.com Interview with:

Demetrios Vavvas, M.D., Ph.D. Co-Director Ocular Regenerative Medical Institute Clinician scientist at Mass. Eye and Ear and Co-Director of the Ocular Regenerative Medicine Institute at Harvard Medical School

Dr. Demetrios Vavvas

Demetrios Vavvas, M.D., Ph.D.
Co-Director Ocular Regenerative Medical Institute
Clinician scientist at Mass. Eye and Ear and Co-Director of the Ocular Regenerative Medicine Institute at Harvard Medical School

Medical Research: What is the background for this study?

Dr. Vavvas: 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 of  age-related macular degeneration disease and the lack of standardization in statin dosage. We were interested in studying the effects of high-dose statins, similar to those showing regression of atherosclerotic plaques, in age-related macular degeneration.

Medical Research: What are the main findings?

Dr. Vavvas: Here, we present for the first time evidence that treatment with high-dose atorvastatin (80mg) is associated with regression of lipid deposits and improvement in visual acuity, without atrophy or neovascularization, in high-risk age-related macular degeneration patients.

Medical Research: What should clinicians and patients take away from your report?

Dr. Vavvas:

  • High dose lipophilic statin administration was associated with regression of large soft drusen and vision gain in 10/23 age-related macular degeneration patients.
  • Duration of treatment before a positive response was observed was usually 1-1.5 years.
  • Patients on high-dose statin appeared to be protected from progression to “wet” neovascular-AMD.

Cholesterol reduction was similarly drastic in responders and non-responders, which suggests that genetic variation may be important in determine who will benefit and who may not.

Age-related macular degeneration is a heterogeneous disease and that targeting the lipid pathway in the appropriate manner and to the appropriate populations we may have the potential not only to slow down or arrest the disease but all to reverse it.

Picture of the back of the eye showing intermediate age-related macular degeneration: Wikipedia image

Picture of the back of the eye showing intermediate age-related macular degeneration: Wikipedia image

Medical Research: What recommendations do you have for future research as a result of this study?

Dr. Vavvas: The role of lipid metabolism and the “ locally oil-spill hypothesis” in age-related macular degeneration should be given more importance. Future studies should take into account genotype and phenotype subgroups of AMD, as well as dosing, lipophilicity, and potency of the statin tested. We would like to initiate a Pharma sponsored or a NIH U10 sponsored prospective control clinical trial with a large number of patients that will include assessments of multiple variables of visual function (contrast, dark adaptation microperimetry) and genetic analysis as well as metabolic analysis. 

Medical Research: Is there anything else you would like to add?

Dr. Vavvas: Our open-label pilot safety/efficacy study suggests that intensive statin treatment may cause regression of high-risk features of age-related macular degeneration, such as drusen, without progression to advanced AMD and without the vision loss that often accompanies spontaneous disappearance of drusen. These results are consistent with the “locally produced oil-spill” hypothesis13 of AMD pathogenesi

Citation:

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, Joan W. Miller. Regression of some High-Risk Features of Age-Related Macular Degeneration (AMD) in Patients Receiving Intensive Statin Treatment. EBioMedicine, 2016; DOI: 1016/j.ebiom.2016.01.033

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Demetrios Vavvas, M.D., Ph.D (2016). Macular Degeneration May Improve with High Dose Statin Therapy MedicalResearch.com