title: Challenges in the development of glaucoma neuroprotection therapy.
abstract: Glaucoma, a disease of the optic nerve and retina, causes blindness in millions of people worldwide. Currently available therapies for this disease only attempt to reduce intraocular pressure, the major risk factor, without addressing the associated optic neuropathy and retinopathy. Development of glaucoma neuroprotective treatment is therefore a pressing unmet medical need. Unfortunately, many challenges hinder this effort, including an incomplete understanding of the mechanism of pathogenesis, leading to uncertain therapeutic targets and confounded by not yet validated preclinical models. Most importantly, with slow disease progression and a less than ideal endpoint measurement method, clinical trials are necessarily large, lengthy, expensive and, to many, prohibitive. No easy solution is available to overcome these challenges. Increased commitment to basic mechanistic research is an essential foundation for dealing with this problem. Innovations in clinical trials with novel surrogate endpoints, nontraditional study designs and the use of surrogate diseases might shorten the study time, reduce the patient sample size and consequently lower the budgetary hurdle for the development of new therapies.
authors.affiliation: North Texas Eye Research Institute, University of North Texas Health Science Center, 3500 Camp Bowie Blvd, Fort Worth, TX 76107, USA. yang.liu@unthsc.edu
authors.forename: Yang
authors.initials: Y
authors.lastname: Liu
authors.forename: Iok-Hou
authors.initials: IH
authors.lastname: Pang
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D018450:Disease Progression; D055808:Drug Discovery; D005901:Glaucoma / Q000188:drug therapy*; D006801:Humans; D018696:Neuroprotective Agents / Q000627:therapeutic use*
chemical_list: D018696:Neuroprotective Agents