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Gamma irradiation of human platelet lysate: validation of efficacy for pathogen reduction and assessment of impacts on hpl performance
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Poster Abstract Presentations
Table 1. hCT-MSC Manufacturing for Phase 1 Trial Donor
Tissue wt (g)
P0 yield/g
P1 yield/cm^2
P1 dbl time (hr)
P1 # dbls
P2 yield/cm^2
P2 dbl time (hr)
P2 # dbls
GMP-075 GMP-075R GMP-087 GMP-088 GMP-088R Mean
34.4 N/A 40.3 25.9 N/A 33.5
1.88e6 N/A 1.53e6 7.04e5 N/A 1.34e6
38,372 N/A 52,180 32,703 N/A 41,085
27.3 N/A 29.3 33.3 N/A 30.0
5.3 N/A 5.7 5.1 N/A 5.4
30,814 35,465 40,116 29,651 30,523 33,527
33.8 27.9 31.6 34.3 33.8 32.3
5.0 5.2 5.3 4.9 5.0 5.1
time compared to FBS containing media (FBSCM). The cultured ADSCs were shown to be positive for CD73, CD90, and CD105, but negative for CD14, CD34, CD45, and HLA-DR in both FBSCM and SFCDM. Adipogenic differentiation capability was similar in both media groups, but chondrogenic and osteogenc differentiation capabilities of ADSCs cultured in SFCDM were higher than those cultured in FBSCM. ADSCs positive for b-galactosidase were about 3 times higher in FBSCM . These results indicate that culture in SFCDM has an advantage in reducing cellular senescence of ADSCs compared to FBS media. ADSCs cultured in FBSCM showed a significant increase in the formation frequency of nucleoplasmic bridges comparable to those cultured in SFCDM, suggesting that culture using SFCDM is more genetically stable. The mRNA expression levels of ADSCs cultured in FBSCM were significantly increased for KEGG classification of aging, apoptotic process, cell death, immune response and inflammatory response compared to SFCDM. Conclusion: SFCDM can provide higher-speed cell production rate than the classical FBSCM. SFCDM enables the maintenance of higher population homogeneity, genetic stability, and excellent differentiation potency. Taken together, these results suggest that culture using SFCDM provides various advantages through which it is possible to obtain safer, stable, and larger amounts of ADSCs. Fig. 1.
treatment of autism spectrum disorder. For this trial, 12 patients ages 2 11 years were sequentially dosed with 1, 2, or 3 doses of 2 million MSC/kg post thaw administered intravenously every 2 months. A manufacturing process was developed for our GMP facility to produce the required doses in a flexible and reproducible manner. Methods, Results & Conclusion: For the trial, 3 lots of hCT-MSCs were manufactured from cord tissue obtained from 3 term healthy male babies delivered after elective routine C-section. After transporting the cord to the Robertson GMP Laboratory, the tissue was cut and digested in a tissue dissociator. The resulting cell suspension was plated in tissue culture flasks for P0 culture in Prime-XV MSC Expansion XSFM culture medium (Irvine Scientific) supplemented with 1% platelet lysate (PL). After harvest, P0 cells were cryopreserved, with a subset of cells expanded for P1 in XSFM without PL, and with a larger cryopreservation and expansion process for P2. For both P1 and P2, cells were seeded at 1000/cm2. Cells at the end of P2 were frozen in multi-compartment cryo-bags at the appropriate concentration for final dose administration. Prior to release to the patient, each lot of P2 hCT-MSC was characterized by flow cytometry for typical MSC markers, tri-lineage differentiation, and the ability to suppress T-cell proliferation. Manufacturing process data for each donor are presented in Table 1. For 2 donors, additional P2 cells were produced from P1 vials during the course of the trial (e.g., “remanufactured” lots) when the original batch was not enough to meet demand. We calculated that after P0 the cells underwent 10.5 doublings on average after the end of P2, with a mean doubling time in P1 P2 of 31.2 hours. The final thawed and prepared P2 cell product had a mean viability of 75% prior to administration to the patient. Preparations are underway for a Phase II clinical trial and the subsequent need for a larger scale and more efficient manufacturing process. We are developing a streamlined process using closed systems for cell expansion, concentration, and cryopreservation of the MSC product that is semi-automated and will minimize the use of clean room facilities. Comparability of the MSC product using the new process will be confirmed prior to use in the Phase II trial. Table 1.
Figure 1. Comparison of population double time and accumulated cell number
273 COMPARATIVE ANALYSIS OF FBS CONTAINING MEDIA AND SERUM FREE CHEMICALLY DEFINED MEDIA FOR ADIPOSE DERIVED STEM CELLS PRODUCTION J. Lee1, M. Kang1, J. Choi1, Y. Chun1, J. Jang1, Y. Yang1, J. Lee1, S. Kim1 & S. Park2 1 R&D center, Xcell Therapeutics, Seoul, Korea (the Republic of), 2Pharmacy, Ajou University, Suwon, Korea (the Republic of)
274 GAMMA IRRADIATION OF HUMAN PLATELET LYSATE: VALIDATION OF EFFICACY FOR PATHOGEN REDUCTION AND ASSESSMENT OF IMPACTS ON HPL PERFORMANCE C. Huang1, F. Liang1, Y. Lin1, Y. Chen2, R. Tseng12 & M. Huang1,2 1 Research & Development, AventaCell Biomedical Corp. Ltd., New Taipei City, Taiwan, 2Research & Development Center, AventaCell BioMedical Corp., Atlanta, Georgia, United States
Background & Aim: For the successful development of production effective stem cell therapy, mass production of consistent quality cells is required. Excellent culture medium is important in mass production of quality cells. Classically, fetal bovine serum (FBS) has been used as culture supplement for stem cells. However, due to the undefined and heterologous composition of FBS, researchers have transitioned to more chemically defined serum-free media. In this study, we aimed to investigate and compare the stem cell characteristics cultured in both media as well as media performance tests. Methods, Results & Conclusion Methods: Media performance was compared with cell growth rate, accumulated cell number, population homogeneity and cell viability. Comparative analysis of adipose derived mesenchymal stem cells (ADSCs) was performed based on stem cell surface marker expression, differentiation potency, genetic stability, senescence analysis, mRNA expression change, and protein expression change. Results: Culture using SFCDM provided more stable population doubling time (PDT), more homogenous cell population, and more cells in a shorter
Background & Aim: Human platelet lysate (hPL) is increasingly popular as a xenogenic-free alternative to FBS for manufacturing cell products. UltraGROTM series products are manufactured from pooled human platelets collected from healthy donors at FDA-licensed, AABB-accredited blood centers. Each donor has been interviewed, evaluated, and donations are tested using FDA-approved methods for infectious diseases screening. Despite the low risk associated with the qualified transfusable platelet units, transmission of infectious agents remains a consideration for human platelet-derived products. In recent European Pharmacopoeia (Ph. Eur.) 9th Edition 5.2.12, implementation of a pathogen reduction procedure in the manufacturing process is suggested. Gamma irradiation is one of the most widely employed methods for pathogen reduction and commercial gamma sterilization facilities are easily accessible. The whole system for irradiated FBS has been well established and applied in clinical trials. Nevertheless, many research articles have addressed the optimal conditions for utilizing gamma irradiation in human plasma and blood components. With these comprehensive references, we previously
Poster Abstract Presentations assessed the feasibility of using gamma irradiation to obtain pathogen-reduced hPL and reported low impacts on the potency for cell expansion. Methods, Results & Conclusion: In this study, we validated the efficacy of gamma irradiation for virus inactivation. Four model viruses (BVDV, Reo3, HSV1, MMV) were chosen, per ICH/EMA guidelines, to represent a range of viruses with different genome, structure, size, and sensitivity to various chemical and physical agents. The virus spiked hPLs were gamma irradiated and the mean values of viral titers showed over 4 log10 reductions across all model viruses. The results demonstrated gamma irradiation is an effective viral reduction procedure for hPL. To assess the impacts of gamma irradiation on the long-term stability of hPL performance, we analyzed UltraGROTM GI series up to one year after gamma irradiation. The results showed growth factors still retained comparable levels to the non-irradiated hPLs. Mesenchymal stromal cells (MSC) cultured with gamma-irradiated hPLs for more than three passages showed similar profiles as with the corresponding non-irradiated hPLs in respect of growth rate, morphology, immunophenotype, trilineage differentiation potency, and immunosuppressive properties. 275 ENHANCE HEPATIC DIFFERENTIATION OF HUMAN WHARTON’S JELLY DERIVED MESENCHYMAL STROMAL CELLS BY USING SODIUM BUTYRATE PRE-TREATED W. Panta1, T. Yoisungnern1, S. Imsoonthornruksa1, S. Suksaweang2, M. Ketudat-Cairns1 & R. Parnpai1 1 Embryo Technology and Stem Cell Research Center and School of Biotechnology, Agricultural Technology, Nakhon Ratchasima, Thailand, 2 Pathology and Laboratory Medicine, Institute of Medicine, Suranaree University of Technology, Muang, Nakhon Ratchasima, Thailand Background & Aim: Human Wharton’s jelly derived mesenchymal stromal cells (hWJ MSCs) of umbilical cord tissue are a richest and more attractive source for stem cell based therapy. Despite of their ability to undergo selfrenewal, they also differentiate into all mesenchyme and some non mesenchyme tissues, including hepatocytes. This study, we differentiated hWJ MSCs into hepatocyte like cells by using sodium butyrate (NaB) pre treated with epidermal growth factor (EGF) and basic fibroblast growth factor (bFGF) supplementation. Methods, Results & Conclusion: Hepatic differentiation of hWJ MSCs was induced with 3 step differentiation protocol. The differentiated cells were examined for the expression of hepatic specific markers and hepatocyte function by using immunocytochemistry, real time reverse transcriptase polymerase chain reaction (RT PCR) and Periodic acid Schiff (PAS) staining. Before hepatic differentiation of hWJ MSCs, we identified embryonic definitive endodermal cells by using RT PCR, which increase of CXCR4 mRNA level, after NaB pre treated along with EGF and bFGF supplementation. Hepatic differentiation of hWJ MSCs by using NaB pre treated along with EGF and bFGF supplementation, the differentiated hWJ MSCs performed high hepatic specific markers at gene and protein levels, and increased signal of glycogen storage accumulation. Our result indicate that hWJ MSCs are able to differentiate into hepatocyte like cells by inducing of hepatic differentiation medium and NaB pre treated with EGF and bFGF supplementation. Therefore, the differentiated hWJ MSCs to hepatocyte like cells by inducing of NaB pre treated with EGF and bFGF condition could represent an alternative source of end stage of liver diseases. 276 SAFETY ISSUES OF PERIBULBAR INJECTION OF UMBILICAL CORD MESENCHYMAL STEM CELL (UC-MSC) IN PATIENTS WITH RETINITIS PIGMENTOSA C. Mangunsong12, B. Putera3, R. Haifa3, M. Suwandjaja2, A. Sharina1, M.B. Sasongko2 & Y.W. Wirohadidjojo2 1 JEC Hospital, Tangerang, Indonesia, 2Gadjah Mada University, Yogyakarta, Yogyakarta, Indonesia, 3Prodia Stemcell Indonesia, Jakarta, Indonesia 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
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(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.
1 hour and 24 hours after injection the cells
1 hour and 24 hours after injection the cells 277 CARTILAGE REGENERATION BY AUTOLOGOUS ADIPOSEDERIVED MESENCHYMAL STEM CELLS FOR THE TREATMENT OF OSTEOARTHRITIS Y. Jiang1, S. Iwata2, C. Yang1, K. Shirakawa3,1 & T. Matsuoka4 1 R&D, STEMCELL Co., Ltd., Tokyo, Japan, 2Riso Clinic, Tokyo, Japan, 3 Center for Southeast Asian Studies, Kyoto University, Kyoto, Japan, 4CEO, STEMCELL Co., Ltd., Tokyo, Japan Background & Aim: Osteoarthritis (OA) is a degenerative joint disease mainly caused by wear and tear of the cartilage cushion between joints.
Poster Abstract Presentations
Table 1. hCT-MSC Manufacturing for Phase 1 Trial Donor
Tissue wt (g)
P0 yield/g
P1 yield/cm^2
P1 dbl time (hr)
P1 # dbls
P2 yield/cm^2
P2 dbl time (hr)
P2 # dbls
GMP-075 GMP-075R GMP-087 GMP-088 GMP-088R Mean
34.4 N/A 40.3 25.9 N/A 33.5
1.88e6 N/A 1.53e6 7.04e5 N/A 1.34e6
38,372 N/A 52,180 32,703 N/A 41,085
27.3 N/A 29.3 33.3 N/A 30.0
5.3 N/A 5.7 5.1 N/A 5.4
30,814 35,465 40,116 29,651 30,523 33,527
33.8 27.9 31.6 34.3 33.8 32.3
5.0 5.2 5.3 4.9 5.0 5.1
time compared to FBS containing media (FBSCM). The cultured ADSCs were shown to be positive for CD73, CD90, and CD105, but negative for CD14, CD34, CD45, and HLA-DR in both FBSCM and SFCDM. Adipogenic differentiation capability was similar in both media groups, but chondrogenic and osteogenc differentiation capabilities of ADSCs cultured in SFCDM were higher than those cultured in FBSCM. ADSCs positive for b-galactosidase were about 3 times higher in FBSCM . These results indicate that culture in SFCDM has an advantage in reducing cellular senescence of ADSCs compared to FBS media. ADSCs cultured in FBSCM showed a significant increase in the formation frequency of nucleoplasmic bridges comparable to those cultured in SFCDM, suggesting that culture using SFCDM is more genetically stable. The mRNA expression levels of ADSCs cultured in FBSCM were significantly increased for KEGG classification of aging, apoptotic process, cell death, immune response and inflammatory response compared to SFCDM. Conclusion: SFCDM can provide higher-speed cell production rate than the classical FBSCM. SFCDM enables the maintenance of higher population homogeneity, genetic stability, and excellent differentiation potency. Taken together, these results suggest that culture using SFCDM provides various advantages through which it is possible to obtain safer, stable, and larger amounts of ADSCs. Fig. 1.
treatment of autism spectrum disorder. For this trial, 12 patients ages 2 11 years were sequentially dosed with 1, 2, or 3 doses of 2 million MSC/kg post thaw administered intravenously every 2 months. A manufacturing process was developed for our GMP facility to produce the required doses in a flexible and reproducible manner. Methods, Results & Conclusion: For the trial, 3 lots of hCT-MSCs were manufactured from cord tissue obtained from 3 term healthy male babies delivered after elective routine C-section. After transporting the cord to the Robertson GMP Laboratory, the tissue was cut and digested in a tissue dissociator. The resulting cell suspension was plated in tissue culture flasks for P0 culture in Prime-XV MSC Expansion XSFM culture medium (Irvine Scientific) supplemented with 1% platelet lysate (PL). After harvest, P0 cells were cryopreserved, with a subset of cells expanded for P1 in XSFM without PL, and with a larger cryopreservation and expansion process for P2. For both P1 and P2, cells were seeded at 1000/cm2. Cells at the end of P2 were frozen in multi-compartment cryo-bags at the appropriate concentration for final dose administration. Prior to release to the patient, each lot of P2 hCT-MSC was characterized by flow cytometry for typical MSC markers, tri-lineage differentiation, and the ability to suppress T-cell proliferation. Manufacturing process data for each donor are presented in Table 1. For 2 donors, additional P2 cells were produced from P1 vials during the course of the trial (e.g., “remanufactured” lots) when the original batch was not enough to meet demand. We calculated that after P0 the cells underwent 10.5 doublings on average after the end of P2, with a mean doubling time in P1 P2 of 31.2 hours. The final thawed and prepared P2 cell product had a mean viability of 75% prior to administration to the patient. Preparations are underway for a Phase II clinical trial and the subsequent need for a larger scale and more efficient manufacturing process. We are developing a streamlined process using closed systems for cell expansion, concentration, and cryopreservation of the MSC product that is semi-automated and will minimize the use of clean room facilities. Comparability of the MSC product using the new process will be confirmed prior to use in the Phase II trial. Table 1.
Figure 1. Comparison of population double time and accumulated cell number
273 COMPARATIVE ANALYSIS OF FBS CONTAINING MEDIA AND SERUM FREE CHEMICALLY DEFINED MEDIA FOR ADIPOSE DERIVED STEM CELLS PRODUCTION J. Lee1, M. Kang1, J. Choi1, Y. Chun1, J. Jang1, Y. Yang1, J. Lee1, S. Kim1 & S. Park2 1 R&D center, Xcell Therapeutics, Seoul, Korea (the Republic of), 2Pharmacy, Ajou University, Suwon, Korea (the Republic of)
274 GAMMA IRRADIATION OF HUMAN PLATELET LYSATE: VALIDATION OF EFFICACY FOR PATHOGEN REDUCTION AND ASSESSMENT OF IMPACTS ON HPL PERFORMANCE C. Huang1, F. Liang1, Y. Lin1, Y. Chen2, R. Tseng12 & M. Huang1,2 1 Research & Development, AventaCell Biomedical Corp. Ltd., New Taipei City, Taiwan, 2Research & Development Center, AventaCell BioMedical Corp., Atlanta, Georgia, United States
Background & Aim: For the successful development of production effective stem cell therapy, mass production of consistent quality cells is required. Excellent culture medium is important in mass production of quality cells. Classically, fetal bovine serum (FBS) has been used as culture supplement for stem cells. However, due to the undefined and heterologous composition of FBS, researchers have transitioned to more chemically defined serum-free media. In this study, we aimed to investigate and compare the stem cell characteristics cultured in both media as well as media performance tests. Methods, Results & Conclusion Methods: Media performance was compared with cell growth rate, accumulated cell number, population homogeneity and cell viability. Comparative analysis of adipose derived mesenchymal stem cells (ADSCs) was performed based on stem cell surface marker expression, differentiation potency, genetic stability, senescence analysis, mRNA expression change, and protein expression change. Results: Culture using SFCDM provided more stable population doubling time (PDT), more homogenous cell population, and more cells in a shorter
Background & Aim: Human platelet lysate (hPL) is increasingly popular as a xenogenic-free alternative to FBS for manufacturing cell products. UltraGROTM series products are manufactured from pooled human platelets collected from healthy donors at FDA-licensed, AABB-accredited blood centers. Each donor has been interviewed, evaluated, and donations are tested using FDA-approved methods for infectious diseases screening. Despite the low risk associated with the qualified transfusable platelet units, transmission of infectious agents remains a consideration for human platelet-derived products. In recent European Pharmacopoeia (Ph. Eur.) 9th Edition 5.2.12, implementation of a pathogen reduction procedure in the manufacturing process is suggested. Gamma irradiation is one of the most widely employed methods for pathogen reduction and commercial gamma sterilization facilities are easily accessible. The whole system for irradiated FBS has been well established and applied in clinical trials. Nevertheless, many research articles have addressed the optimal conditions for utilizing gamma irradiation in human plasma and blood components. With these comprehensive references, we previously
Poster Abstract Presentations assessed the feasibility of using gamma irradiation to obtain pathogen-reduced hPL and reported low impacts on the potency for cell expansion. Methods, Results & Conclusion: In this study, we validated the efficacy of gamma irradiation for virus inactivation. Four model viruses (BVDV, Reo3, HSV1, MMV) were chosen, per ICH/EMA guidelines, to represent a range of viruses with different genome, structure, size, and sensitivity to various chemical and physical agents. The virus spiked hPLs were gamma irradiated and the mean values of viral titers showed over 4 log10 reductions across all model viruses. The results demonstrated gamma irradiation is an effective viral reduction procedure for hPL. To assess the impacts of gamma irradiation on the long-term stability of hPL performance, we analyzed UltraGROTM GI series up to one year after gamma irradiation. The results showed growth factors still retained comparable levels to the non-irradiated hPLs. Mesenchymal stromal cells (MSC) cultured with gamma-irradiated hPLs for more than three passages showed similar profiles as with the corresponding non-irradiated hPLs in respect of growth rate, morphology, immunophenotype, trilineage differentiation potency, and immunosuppressive properties. 275 ENHANCE HEPATIC DIFFERENTIATION OF HUMAN WHARTON’S JELLY DERIVED MESENCHYMAL STROMAL CELLS BY USING SODIUM BUTYRATE PRE-TREATED W. Panta1, T. Yoisungnern1, S. Imsoonthornruksa1, S. Suksaweang2, M. Ketudat-Cairns1 & R. Parnpai1 1 Embryo Technology and Stem Cell Research Center and School of Biotechnology, Agricultural Technology, Nakhon Ratchasima, Thailand, 2 Pathology and Laboratory Medicine, Institute of Medicine, Suranaree University of Technology, Muang, Nakhon Ratchasima, Thailand Background & Aim: Human Wharton’s jelly derived mesenchymal stromal cells (hWJ MSCs) of umbilical cord tissue are a richest and more attractive source for stem cell based therapy. Despite of their ability to undergo selfrenewal, they also differentiate into all mesenchyme and some non mesenchyme tissues, including hepatocytes. This study, we differentiated hWJ MSCs into hepatocyte like cells by using sodium butyrate (NaB) pre treated with epidermal growth factor (EGF) and basic fibroblast growth factor (bFGF) supplementation. Methods, Results & Conclusion: Hepatic differentiation of hWJ MSCs was induced with 3 step differentiation protocol. The differentiated cells were examined for the expression of hepatic specific markers and hepatocyte function by using immunocytochemistry, real time reverse transcriptase polymerase chain reaction (RT PCR) and Periodic acid Schiff (PAS) staining. Before hepatic differentiation of hWJ MSCs, we identified embryonic definitive endodermal cells by using RT PCR, which increase of CXCR4 mRNA level, after NaB pre treated along with EGF and bFGF supplementation. Hepatic differentiation of hWJ MSCs by using NaB pre treated along with EGF and bFGF supplementation, the differentiated hWJ MSCs performed high hepatic specific markers at gene and protein levels, and increased signal of glycogen storage accumulation. Our result indicate that hWJ MSCs are able to differentiate into hepatocyte like cells by inducing of hepatic differentiation medium and NaB pre treated with EGF and bFGF supplementation. Therefore, the differentiated hWJ MSCs to hepatocyte like cells by inducing of NaB pre treated with EGF and bFGF condition could represent an alternative source of end stage of liver diseases. 276 SAFETY ISSUES OF PERIBULBAR INJECTION OF UMBILICAL CORD MESENCHYMAL STEM CELL (UC-MSC) IN PATIENTS WITH RETINITIS PIGMENTOSA C. Mangunsong12, B. Putera3, R. Haifa3, M. Suwandjaja2, A. Sharina1, M.B. Sasongko2 & Y.W. Wirohadidjojo2 1 JEC Hospital, Tangerang, Indonesia, 2Gadjah Mada University, Yogyakarta, Yogyakarta, Indonesia, 3Prodia Stemcell Indonesia, Jakarta, Indonesia 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
S83
(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.
1 hour and 24 hours after injection the cells
1 hour and 24 hours after injection the cells 277 CARTILAGE REGENERATION BY AUTOLOGOUS ADIPOSEDERIVED MESENCHYMAL STEM CELLS FOR THE TREATMENT OF OSTEOARTHRITIS Y. Jiang1, S. Iwata2, C. Yang1, K. Shirakawa3,1 & T. Matsuoka4 1 R&D, STEMCELL Co., Ltd., Tokyo, Japan, 2Riso Clinic, Tokyo, Japan, 3 Center for Southeast Asian Studies, Kyoto University, Kyoto, Japan, 4CEO, STEMCELL Co., Ltd., Tokyo, Japan Background & Aim: Osteoarthritis (OA) is a degenerative joint disease mainly caused by wear and tear of the cartilage cushion between joints.