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

191 Citations (Scopus)

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
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

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Geographic Atrophy
Ciliary Neurotrophic Factor
Macular Degeneration
Visual Acuity
Technology
Therapeutics

Keywords

  • Apoptosis
  • Photoreceptor degeneration
  • Retina

ASJC Scopus subject areas

  • General

Cite this

Ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration. / Zhang, Kang; Hopkins, Jill J.; Heier, Jeffrey S.; Birch, David G.; Halperin, Lawrence S.; Albini, Thomas A; Brown, David M.; Jaffe, Glenn J.; Taoj, Weng; Williams, George A.

In: Proceedings of the National Academy of Sciences of the United States of America, Vol. 108, No. 15, 12.04.2011, p. 6241-6245.

Research output: Contribution to journalArticle

Zhang, Kang ; Hopkins, Jill J. ; Heier, Jeffrey S. ; Birch, David G. ; Halperin, Lawrence S. ; Albini, Thomas A ; Brown, David M. ; Jaffe, Glenn J. ; Taoj, Weng ; Williams, George A. / Ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration. In: Proceedings of the National Academy of Sciences of the United States of America. 2011 ; Vol. 108, No. 15. pp. 6241-6245.
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