title: Ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration.
abstract: There is no treatment available for vision loss associated with advanced dry age-related macular degeneration (AMD) or geographic atrophy (GA). In a pilot, proof of concept phase 2 study, we evaluated ciliary neurotrophic factor (CNTF) delivered via an intraocular encapsulated cell technology implant for the treatment of GA. We designed a multicenter, 1-y, double-masked, sham-controlled dose-ranging study. Patients with GA were randomly assigned to receive a high-or low-dose implant or sham surgery. The primary endpoint was the change in best corrected visual acuity (BCVA) at 12 mo. CNTF treatment resulted in a dose-dependent increase in retinal thickness. This change was followed by visual acuity stabilization (loss of less than 15 letters) in the high-dose group (96.3%) compared with low-dose (83.3%) and sham (75%) group. A subgroup analysis of those with baseline BCVA at 20/63 or better revealed that 100% of patients in the high-dose group lost <15 letters compared with 55.6% in the combined low-dose/sham group (P = 0.033). There was a 0.8 mean letter gain in the high-dose group compared with a 9.7 mean letter loss in the combined low-dose/sham group (P = 0.0315). Both the implant and the implant procedure were well-tolerated. These findings suggest that CNTF delivered by the encapsulated cell technology implant appears to slow the progression of vision loss in GA, especially in eyes with 20/63 or better vision at baseline.
authors: Shiley Eye Center and Institute for Genomic Medicine, University of California at San Diego, La Jolla, CA 92093, USA. kang.zhang@gmail.com
authors: Kang
authors: K
authors: Zhang
authors: Jill J
authors: JJ
authors: Hopkins
authors: Jeffrey S
authors: JS
authors: Heier
authors: David G
authors: DG
authors: Birch
authors: Lawrence S
authors: LS
authors: Halperin
authors: Thomas A
authors: TA
authors: Albini
authors: David M
authors: DM
authors: Brown
authors: Glenn J
authors: GJ
authors: Jaffe
authors: Weng
authors: W
authors: Tao
authors: George A
authors: GA
authors: Williams
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D002214:Capsules; D020934:Ciliary Neurotrophic Factor / Q000008:administration & dosage*; D004343:Drug Implants / Q000008:administration & dosage*; D005260:Female; D006801:Humans; D008268:Macular Degeneration / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D012160:Retina / Q000187:drug effects* / Q000473:pathology; D016896:Treatment Outcome; D014785:Vision, Ocular / Q000187:drug effects
chemical_list: D002214:Capsules; D020934:Ciliary Neurotrophic Factor; D004343:Drug Implants