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

Kang Zhang, Jill J. Hopkins, Jeffrey S. Heier, David G. Birch, Lawrence S. Halperin, Thomas A. Albini, David M. Brown, Glenn J. Jaffe, Weng Taoj, George A. Williams

Research output: Contribution to journalArticle

201 Scopus citations

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 doseranging 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.

Original languageEnglish (US)
Pages (from-to)6241-6245
Number of pages5
JournalProceedings of the National Academy of Sciences of the United States of America
Volume108
Issue number15
DOIs
StatePublished - Apr 12 2011

Keywords

  • Apoptosis
  • Photoreceptor degeneration
  • Retina

ASJC Scopus subject areas

  • General

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