Abstract
Purpose :
To evaluate the safety and efficacy of Retinal pigment epithelium (RPE) transplantation for retinal degenerative diseases affecting the RPE-photoreceptor complex, clinically to treat the AMD and Stargardt’s disease.
Methods :
The xeno-free clinical-grade human embryonic stem cell line (CTS hESC) was induced to RPE cells by spontaneous differentiation method. A serious of safety assessments were finished for potential bacteria, mycoplasma, murine viruses according to standard protocols. The characteristics of this CTS hESC derived RPE-specific attributes including purity, differentiation and phagocytosis by quantitative PCR, FACS and immunohistochemistry staining. The purified CTS hESC derived RPE at passage 3 were used for clinical purpose. After registered with ClinicalTrials.gov (ChiCTR-OCB-15006423), we performed clinical study of subretinal transplantation of CTS hESC derived RPE in treatment of wet Age-related Macular Degeneration (wAMD).
Results :
The first three patients underwent vitrectomy and removal of choroidal neovascularization (CNV) membrane successfully. During the three months follow-up till now, there were no rejection showed in the clinical tests, interestingly all three patients had improved their vision 7, 15 to 41 letters respectively in ETDRS visual acuity test and the patients’ previous feeling of central vision blurry due to CNV disappeared.
Conclusions :
As we know this is the first study of subretinal transplantation of hESC derived RPE in the treatment of wet Age-related Macular Degeneration, which opened a new approach for the treatment of retinal degeneration diseases by the human embryonicstem cells in clinics.
This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.