title: Challenges in the Development of Therapy for Dry Age-Related Macular Degeneration.
abstract: Dry age-related macular degeneration (AMD), a multifactorial progressive degenerative disease of the retinal photoreceptors, pigmented epithelium and Bruch's membrane/choroid in central retina, causes visual impairment in millions of elderly people worldwide. The only available therapy for this disease is the over-the-counter (OTC) multi-vitamins plus macular xanthophyll (lutein/zeaxanthin) which attempts to block the damages of oxidative stress and ionizing blue light. Therefore development of dry AMD prescribed treatment is a pressing unmet medical need. However, this effort is currently hindered by many challenges, including an incomplete understanding of the mechanism of pathogenesis that leads to uncertain targets, confounded by not yet validated preclinical models and the difficulty to deliver the drugs to the posterior segment of the eye. Additionally, with slow disease progression and a less than ideal endpoint measurement method, clinical trials are necessarily large, lengthy and expensive. Increased commitment to research and development is an essential foundation for dealing with these problems. Innovations in clinical trials with novel endpoints, nontraditional study designs and the use of surrogate diseases might shorten the study time, reduce the patient sample size and consequently lower the budget for the development of the new therapies for the dry AMD.
authors.affiliation: Department of Ophthalmology, University of Texas Southwestern Medical Center, 75390, Dallas, TX, USA. Cynthua.Wei@UTSouthwestern.edu.
authors.forename: Cynthia X
authors.initials: CX
authors.lastname: Wei
authors.affiliation: Departments of Biological Science, Allergan Inc, RD3-2B, 2525 Dupont Drive, Irvine, CA, 92612, USA. Sun_Aixu@Allergan.com.
authors.forename: Aixu
authors.initials: A
authors.lastname: Sun
authors.affiliation: Departments of Biological Science, Allergan Inc, RD3-2B, 2525 Dupont Drive, Irvine, CA, 92612, USA. Yu_Ying@Allergan.com.
authors.forename: Ying
authors.initials: Y
authors.lastname: Yu
authors.affiliation: Departments of Biological Science, Allergan Inc, RD3-2B, 2525 Dupont Drive, Irvine, CA, 92612, USA. Liu_Qianyong@Allergan.com.
authors.forename: Qianyong
authors.initials: Q
authors.lastname: Liu
authors.affiliation: Department of Community Medicine, University of North Texas Health Science Center, Fort Worth, TX, 76107, USA. Yue-Qing.Tan@unthsc.edu.
authors.forename: Yue-Qing
authors.initials: YQ
authors.lastname: Tan
authors.affiliation: Department of Ophthalmology, University of Texas Southwestern Medical Center, 75390, Dallas, TX, USA. Isamu.Tachibana@UTSouthwestern.edu.
authors.forename: Isamu
authors.initials: I
authors.lastname: Tachibana
authors.affiliation: University of Houston College of Optometry, Houston, TX, 77204, USA. hzeng.2017@alumni.opt.uh.edu.
authors.forename: Hong
authors.initials: H
authors.lastname: Zeng
authors.affiliation: Departments of Biological Science, Allergan Inc, RD3-2B, 2525 Dupont Drive, Irvine, CA, 92612, USA. Jiye_wei@yahoo.com.
authors.forename: Ji-Ye
authors.initials: JY
authors.lastname: Wei
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D004358:Drug Therapy / Q000379:methods / Q000639:trends; D020022:Genetic Predisposition to Disease / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods; D057092:Geographic Atrophy / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D008810:Mice, Inbred C57BL; D009154:Mutation; D033581:Stem Cell Transplantation / Q000379:methods
keywords: Anti-VEGF DARPin; Antioxidants; Bruch’s membrane; Complement; Drusen; Dry AMD; Geographic atrophy; OZURDEX; Photoreceptor; Retinal pigment epithelium