Safety issues of peribulbar injection of umbilical cord mesenchymal stem cell (UC-MSC) in patients with retinitis pigmentosa

Abstract

Background & Aim Hereditary retinal dystrophies, specifically retinitis pigmentosa (RP) are clinically and genetically heterogeneous diseases affecting primarily retinal cells and retinal pigment epithelial cells with blindness as a final outcome1. Retinitis pigmentosa is the term given to a set of hereditary retinal diseases that feature degeneration of rod and cone photoreceptors2. Typical symptoms include night blindness followed by decreasing visual fields, leading to tunnel vision and eventually legal blindness or, in many cases, complete blindness3. The worldwide prevalence of retinitis pigmentosa is about 1 in 4000 for a total of more than 1 million aff ected individuals. The disease can be inherited as an autosomal-dominant (about 30–40% of cases), auto somal-recessive (50–60%), or X-linked (5–15%) trait4. This disease is no hope no option treatment. Recent preclinical studies and clinical trials suggest cells-based therapy for the treatment of retinal degenerative diseases5. Cell-based therapy seems to be a promising strategy in Retinitis Pigmenotosa as it has the potential to regenerate new photoreceptors or retinal pigment epithelial (RPE) cells. Several types of stem cells had been investigated. However, in vivo studies showed that the cells from human umbilical corfd tissue appears to be the most effective in many diseases6-7 The purpose of this study is to evaluate of the safety of peribulbar injection of umbilical cord mesenchymal stem cells in patients with retinitis pigmentosa by a prospective, multi-center, randomized clinical trial. Methods, Results & Conclusion Methods This study was conducted on 18 eyes of 18 volunteers with retinitis pigmentosa, The cells delivery with peribulbar injection method. Clinical examinitation, visus and fundus examination, multifocal electroretinography were performed before and after an peribulbar injection of approximately one-million UC-MSC. The patients were followed in 1 hour, 24 hours and 1 month for safety evaluation. Results: There are adverse events, like edema under the eyes, were observed in eyes all patients 1 hour after transplantation of UC-MSCs. The edema will disappear within 24 hours. These patients reported improvements in perception of the light and visus after one week Conclusion Peribulbar injection of umbilical cord mesenchymal stem cells in eyes in patient with retinitis pigmentosa can occur edema under the eye which disappear within 24 hours. Hereditary retinal dystrophies, specifically retinitis pigmentosa (RP) are clinically and genetically heterogeneous diseases affecting primarily retinal cells and retinal pigment epithelial cells with blindness as a final outcome1. Retinitis pigmentosa is the term given to a set of hereditary retinal diseases that feature degeneration of rod and cone photoreceptors2. Typical symptoms include night blindness followed by decreasing visual fields, leading to tunnel vision and eventually legal blindness or, in many cases, complete blindness3. The worldwide prevalence of retinitis pigmentosa is about 1 in 4000 for a total of more than 1 million aff ected individuals. The disease can be inherited as an autosomal-dominant (about 30–40% of cases), auto somal-recessive (50–60%), or X-linked (5–15%) trait4. This disease is no hope no option treatment. Recent preclinical studies and clinical trials suggest cells-based therapy for the treatment of retinal degenerative diseases5. Cell-based therapy seems to be a promising strategy in Retinitis Pigmenotosa as it has the potential to regenerate new photoreceptors or retinal pigment epithelial (RPE) cells. Several types of stem cells had been investigated. However, in vivo studies showed that the cells from human umbilical corfd tissue appears to be the most effective in many diseases6-7 The purpose of this study is to evaluate of the safety of peribulbar injection of umbilical cord mesenchymal stem cells in patients with retinitis pigmentosa by a prospective, multi-center, randomized clinical trial. This study was conducted on 18 eyes of 18 volunteers with retinitis pigmentosa, The cells delivery with peribulbar injection method. Clinical examinitation, visus and fundus examination, multifocal electroretinography were performed before and after an peribulbar injection of approximately one-million UC-MSC. The patients were followed in 1 hour, 24 hours and 1 month for safety evaluation.