title: Pegaptanib sodium for ocular vascular disease.
abstract: Pegaptanib sodium (Macugen) is a selective RNA aptamer that inhibits vascular endothelial growth factor (VEGF) 165 , the VEGF isoform primarily responsible for pathologic ocular neovascularization and vascular permeability, while sparing the physiological isoform VEGF 121 . After more than 10 years in development and preclinical study, pegaptanib was shown in clinical trials to be effective in treating choroidal neovascularization associated with age-related macular degeneration. Its excellent ocular and systemic safety profile has also been confirmed in patients receiving up to three years of therapy. Early, well-controlled studies further suggest that pegaptanib may provide therapeutic benefit for patients with diabetic macular edema, proliferative diabetic retinopathy and retinal vein occlusion. Notably, pegaptanib was the first available aptamer approved for therapeutic use in humans and the first VEGF inhibitor available for the treatment of ocular vascular diseases.
authors.affiliation: Retina-Vitreous Service, Aravind Eye Hospital and Postgraduate Institute, Anna Nagar, Madurai, India.
authors.forename: Dhananjay
authors.initials: D
authors.lastname: Shukla
authors.forename: Perumalsamy
authors.initials: P
authors.lastname: Namperumalsamy
authors.forename: Mauro
authors.initials: M
authors.lastname: Goldbaum
authors.forename: Emmett T
authors.initials: ET
authors.lastname: Cunningham
mesh_terms: D000818:Animals; D052157:Aptamers, Nucleotide / Q000627:therapeutic use*; D006801:Humans; D012164:Retinal Diseases / Q000188:drug therapy* / Q000378:metabolism; D016896:Treatment Outcome; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors / Q000378:metabolism
chemical_list: D052157:Aptamers, Nucleotide; D042461:Vascular Endothelial Growth Factor A; C495058:pegaptanib