Abstract
Purpose :
To evaluate the effect of subretinally-transplanted Human Central Nervous System Stem cells (HuCNS-SC) on progression of geographic atrophy (GA) in subjects with non-neovascular age related macular degeneration (NNVAMD).
Methods :
Twenty two eyes of 11 subjects with GA were enrolled in this IRB-approved, multicenter prospective clinical trial (NCD:CL-N01-AMD). To be included in the study, subjects were required to have bilateral GA due to AMD only, and one eye (study eye) was selected for treatment. For this analysis, the transplanted eye was considered to be the study eye and fellow eye was treated as a control. A total of 1.0 X 106 HuCNS-SC cells in a volume of 0.02mL were directly infused into the subretinal space superotemporal to the fovea near the junctional zone outside the area of GA. All subjects underwent Fundus Autofluorescence imaging using the Spectralis HRA+OCT (Heidelberg Engineering Inc.,Germany). Total GA area in both eyes was measured at baseline and month 12 by certified reading center graders using the Spectralis Region Finder software. Sectoral (clock hour)/ directional GA progression rates with respect to the foveal center in both eyes were also calculated using the polar transformation method in Image J. Total GA area and sectoral GA progression rates were compared in both study and control eyes using Wilcoxon Signed Ranks Test.
Results :
The mean change in total GA area at month 12 was not statistically significantly different between study and fellow eyes (1.07 vs. 2.08 mm2, p=0.08). The month 12 sectoral or directional GA growth rate for the supero-temporal region (i.e. the location of HuCNS-SC cells Transplantation) showed a significantly slower progression rate in study eyes when compared to the same region in fellow eyes (0.06 ± 0.11 vs. 0.21 ± 0.13 mm2,p=0.03,Table 1). The progression rate in the supero-temporal quadrant was also significantly slower than the other three quadrants of the study eye combined (p=0.003)
Conclusions :
In this small pilot study, HuCNS stem cell transplantation appeared to be associated with a slower expansion of the GA lesion in the quadrant in which the therapy was delivered. Larger confirmatory studies are required. Sectoral or directional analysis of growth rates of GA, however, may be a useful approach for assessing the efficacy of locally-delivered therapies.
This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.