Expert panel in macular degeneration recommends paradigm shift for future directions

July 26, 2019

(Boston)-- A panel of investigators assembled by the National Advisory Eye Council (NAEC) calls for large-scale collaborative research to address dry macular degeneration--the leading cause of blindness among the elderly--for which there is currently no effective treatment.

The NAEC--a 12-member panel that helps guide the National Eye Institute--recognized the need to investigate the current state of research on this disease, in order to guide the direction in which to take future research. The article, published in Nature Communications, is the product of a group of scientists tasked by the NAEC to do just that, as well as propose ideas on how to expand knowledge of this disease.

There are two types of age-related macular degeneration (AMD). "Wet" (neovascular) AMD, for which there are effective treatments that target limiting the inappropriate growth of new blood vessels that end up disrupting vision in the delicate tissues of the eye. "Dry" (non-neovascular) AMD is a more gradual vision loss from deposits of a substance called drusen, composed of fats and proteins. The proposed causes of dry AMD are numerous and are not all clear to the scientific and medical community.

"There are a plethora of genetic and environmental factors interweaving and contributing to this disease in ways known and unknown," explained co-corresponding author Lindsay Farrer, PhD, chief of the Biomedical Genetics division at Boston University School of Medicine. "Our article reflects on what we know thus far and calls for interdisciplinary research, with ideas on where to go next, towards answers that will bring about meaningful clinical results."

Among the ideas proposed in the article are drawing forth "integrated collaboration of leading clinicians, imaging experts, a wide variety of basic scientists, bioinformaticians and biostaticians" as well as creating a large biorepository of eye tissue from donors with and without AMD, generating multiple types of 'omics data from disease and normal eye tissue, and designing computer models of the disease. "Success in these areas will likely be achieved most expediently and effectively by promoting collaborative efforts of multidisciplinary investigator teams and developing centralized resources including clinical, imaging, omic, and other types of data as well as carefully phenotyped eye tissue from large cohorts of patients with and without AMD."

The authors also ask agencies such as the National Institutes of Health to reconsider how they allocate funding in order to make this possible. In addition, the authors suggest longer follow-up times with clinical trial patients, given there likely are different factors at play at different stages of the disease.
Funding for this article was provided by NIH EY027691; NIH R42 EY029625-01; BrightFocus Foundation; Macular Degeneration Foundation; Wilmer-Bayer Alliance Grant, Bayer Pharmaceuticals, Inc.; Unrestricted Grant from Research to Prevent Blindness (Wilmer Eye Institute), Robert Bond Welch Professorship. C.B.R.: NIH R01 EY026161; P30 EY005722 to Duke University, a Research to Prevent Blindness (RPB)/International Retinal Research Foundation (IRRF) Catalyst Award for Innovative Research Approaches for AMD, an unrestricted grant from RPB (to the Duke Eye Center), and a Fighting Blindness Individual Investigator Award. A.D.D.: NIHR Biomedical Research Centre Moorfields Eye Hospital and UCL-Institute of Ophthalmology, National Eye Research Centre UK, Macular Society UK, Medical Research Council UK, Rosetrees Trust UK. M.B.G.: Harold and Pauline Price Foundation, Research to Prevent Blindness, NY, NY, NIH/NEI R01 EY09859 Gorin (PI). J.W.M.: NEI Core Facility Grant EY014104, Yeatts Retina Fund, Research to Prevent Blindness, Retina Research Fund, Champalimaud Vision Award. C.A.T.: an unrestricted grant from RPB (to the Duke Eye Center). M.U.: EYE-RISK is funded by the Horizon 2020 program of the European Union. Funding is provided to Marius Ueffing in the period of 2015-2019 under Grant Agreement number 634479. M.Z.: Joseph J. and Marguerite DiSepio Retina Research Fund; Eng Family Foundation; New Jersey Lions Eye Research Foundation; Paid Consultant for: California Institute of Regenerative Medicine, Cell Cure, Chengdu Kanghong Biotechnology Co., Coherus Biosciences, Inc., Daiichi Sankyo, Frequency Therapeutics, Foundation Fighting Blindness, Genentech/Roche, Healios KK, Inc., Iridex, Isarna Therapeutics, Makindus, Novartis Pharma AG, Ophthotech Corp., Percept Corp. L.A.F.: NIH U01-AG032984, NIH UF1-AG046198, NIH R01AG048927, NIH RF1-AG057519.

Editor's Note:

J.T.H.: Grant funding from Bayer Pharmaceuticals, Inc. J.W.M.: Consultant/Advisor for Genentech/Roche, Bausch+Lomb, Kalvista Pharmaceuticals, ONL Therapeutics; Grant Support from Lowy Medical Research Institute; Equity in ONL Therapeutics; Patents/Royalties from ONL Therapeutics/Mass. Eye and Ear, Valeant Pharmaceuticals/Mass. Eye and Ear. C.A.T.: Alcon royalties for surgical technologies. M.Z.: Cell Cure, Chengdu Kanghong Biotech, Coherus Biosciences, Daiichi Sankyo, Frequency Therapeutics, Healios KK, Iridex, Isarna Therapeutics, Genentech/Roche, Makindus, Novartis Pharma AG, Ophthotech, Percept Corp., Rutgers University (patent). The remaining authors declare no competing interests.

Boston University School of Medicine

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