Abstract
Stem cell therapy may provide a safe and promising treatment for retinal diseases. Wet age-related macular degeneration (wet-AMD) is a leading cause of blindness in China. We developed a clinical-grade human embryonic stem cell (hESC) line, Q-CTS-hESC-2, under xeno-free conditions that differentiated into retinal pigment epithelial cells (Q-CTS-hESC-2-RPE). A clinical trial with three wet-AMD patients was initiated in order to study the safety and tolerance to Q-CTS-hESC-2-RPE cell transplants. The choroidal neovascularization membrane was removed and then a suspension of 1 × 106 Q-CTS-hESC-2-RPE cells were injected into a subfoveal pocket. The patients were followed for 12 months during which no adverse effects resulting from the transplant were observed. Anatomical evidence suggested the existence of new RPE-like cell layer in the previously damaged area. Visual and physiological testing indicated limited functional improvement, albeit to different degrees between patients. This study provides some promising early results concerning the use of transplanted hESC-RPE cells to alleviate wet-AMD.
Highlights
Age-related macular degeneration (AMD) is the leading cause of blindness among the elderly in many countries[1]
Human embryonic stem cells (ESCs)-derived retinal pigment epithelium (RPE) Cells transplanted into SCID mice and Royal College of Surgeons (RCS) rats It took ~125 days and three passages to efficiently induce human embryonic stem cell (hESC) to differentiate into RPE cells that were suitable for clinical use (Supplementary Fig. S1a, b)
Fluorescentactivated cell sorting (FACS) analysis indicated that the purity of the hESC-RPE cell cultures was >99%, as indicated by the number of cells labeled positive for RPE markers such as Bestrophin-1 (99.2% ± 0.2%, n = 3), RPE65 (99.3% ± 0.2%, n = 3) and CRALBP (99.2% ± 0.5%, n = 3) (Supplementary Fig. S1e)
Summary
Age-related macular degeneration (AMD) is the leading cause of blindness among the elderly in many countries[1]. The prevalence rates of early (mainly refers to drusen formation in the subretinal space) and late (choroidal neovascularization (CNV) or geographic atrophy occured) AMD in Chinese individuals 50 years of age or older were estimated to be 9.5% and 1.0%, respectively. Wet-AMD predominated, and its medical care has become a major challenge[4,5]. Current treatments, such as the intraocular injection of anti-vascular endothelial growth factor drugs (VEGF), have revolutionized the clinical management of wet-AMD; monthly injections are tedious for patients and only control neovascular lesions. CNV in advanced wetAMD causes substantial damage to the RPE and photoreceptors[6]. Many patients may still lose their vision because of late diagnosis or inadequate treatment[7]
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