Ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration

• DOI: 10.1073/pnas.1018987108
• Publication Date: 2011-03-29T02:34:26Z
• Authors: Kang Zhang; Jill J. Hopkins; Jeffrey S. Heier; David G. Birch; Lawrence S. Halperin; Thomas A. Albini; David M. Brown; Glenn J. Jaffe; Weng Tao; George A. Williams
• Keywords: Ciliary neurotrophic factor; Sham surgery; Clinical endpoint

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 the GA. We designed multicenter, 1-y, double-masked, sham-controlled dose-ranging study. Patients GA were randomly assigned to receive high-or low-dose sham surgery. The primary endpoint was change in best corrected visual acuity (BCVA) at 12 mo. CNTF resulted dose-dependent increase retinal thickness. This followed by stabilization (loss less than 15 letters) high-dose group (96.3%) compared (83.3%) and (75%) group. A subgroup analysis those baseline BCVA 20/63 better revealed that 100% patients lost <15 letters 55.6% combined low-dose/sham ( P = 0.033). 0.8 mean letter gain 9.7 0.0315). Both procedure well-tolerated. These findings suggest appears slow progression GA, especially eyes baseline.