Experience With a Subretinal Cell-based Therapy in Patients With Geographic Atrophy Secondary to Age-related Macular Degeneration

Am J Ophthalmol. 2017 Jul;179:67-80. doi: 10.1016/j.ajo.2017.04.006. Epub 2017 Apr 21.


Purpose: To evaluate the safety and tolerability of and clinical response to a single, subretinal dose of human umbilical tissue-derived cells (palucorcel [CNTO-2476]) in the eyes of adults aged ≥50 years with bilateral geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

Design: Phase 1/2a, multicenter, open-label, dose-escalation, fellow-eye-controlled study.

Methods: In the phase 1 portion, eyes were assigned to receive a single, subretinal dose of palucorcel (ranging from 6.0 × 104 to 5.6 × 105 viable cells). In the phase 2a portion, eyes were assigned to one of 2 palucorcel doses (6.0 × 104 or 3.0 × 105 cells) determined during the phase 1 portion. The intervention eye was the eye with worse baseline visual acuity.

Results: A total of 35 eligible subjects underwent at least a partial surgical procedure. Palucorcel was administered in 33 eyes. Overall, 17.1% (6/35) of subjects experienced retinal detachments and 37.1% (13/35) experienced retinal perforations. No episodes of immune rejection or tumor formation were observed. At 1 year, ≥10- and ≥15-letter gains in best-corrected visual acuity were observed in 34.5% (10/29) and 24.1% (7/29) of eyes receiving palucorcel, respectively, and in 3.3% (1/30; for both) of fellow eyes.

Conclusions: The subretinal delivery procedure in this study was associated with a high rate of retinal perforations (n = 13) and retinal detachments (n = 6). When cells were sequestered in the subretinal space, palucorcel was well tolerated and may be associated with improvements in visual acuity. Larger randomized controlled studies are required to confirm these results. Future studies would require a modified surgical approach.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Aged
  • Aged, 80 and over
  • Cell- and Tissue-Based Therapy / methods*
  • Female
  • Fluorescein Angiography
  • Follow-Up Studies
  • Fundus Oculi
  • Geographic Atrophy / diagnosis
  • Geographic Atrophy / etiology
  • Geographic Atrophy / therapy*
  • Humans
  • Macular Degeneration / complications*
  • Macular Degeneration / diagnosis
  • Macular Degeneration / therapy
  • Male
  • Prospective Studies
  • Retina / pathology*
  • Treatment Outcome
  • Visual Acuity*