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Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism

BACKGROUND: Autism is a pervasive neurodevelopmental disorder. At present there are no defined mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem cell transplantation may offer a unique treatment strategy for autism due to immune and neural dysregulation obser...

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Autores principales: Lv, Yong-Tao, Zhang, Yun, Liu, Min, Qiuwaxi, Jia-na-ti, Ashwood, Paul, Cho, Sungho Charles, Huan, Ying, Ge, Ru-Cun, Chen, Xing-Wang, Wang, Zhao-Jing, Kim, Byung-Jo, Hu, Xiang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3765833/
https://www.ncbi.nlm.nih.gov/pubmed/23978163
http://dx.doi.org/10.1186/1479-5876-11-196
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author Lv, Yong-Tao
Zhang, Yun
Liu, Min
Qiuwaxi, Jia-na-ti
Ashwood, Paul
Cho, Sungho Charles
Huan, Ying
Ge, Ru-Cun
Chen, Xing-Wang
Wang, Zhao-Jing
Kim, Byung-Jo
Hu, Xiang
author_facet Lv, Yong-Tao
Zhang, Yun
Liu, Min
Qiuwaxi, Jia-na-ti
Ashwood, Paul
Cho, Sungho Charles
Huan, Ying
Ge, Ru-Cun
Chen, Xing-Wang
Wang, Zhao-Jing
Kim, Byung-Jo
Hu, Xiang
author_sort Lv, Yong-Tao
collection PubMed
description BACKGROUND: Autism is a pervasive neurodevelopmental disorder. At present there are no defined mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem cell transplantation may offer a unique treatment strategy for autism due to immune and neural dysregulation observed in this disease. This non-randomized, open-label, single center phase I/II trial investigated the safety and efficacy of combined transplantation of human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) in treating children with autism. METHODS: 37 subjects diagnosed with autism were enrolled into this study and divided into three groups: CBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy), Combination group (9 subjects, received both CBMNC and UCMSC transplantation and rehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy). Transplantations included four stem cell infusions through intravenous and intrathecal injections once a week. Treatment safety was evaluated with laboratory examinations and clinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to assess the therapeutic efficacy at baseline (pre-treatment) and following treatment. RESULTS: There were no significant safety issues related to the treatment and no observed severe adverse effects. Statistically significant differences were shown on CARS, ABC scores and CGI evaluation in the two treatment groups compared to the control at 24 weeks post-treatment (p < 0.05). CONCLUSIONS: Transplantation of CBMNCs demonstrated efficacy compared to the control group; however, the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the CBMNC transplantation alone. There were no safety issues noted during infusion and the whole monitoring period. TRIAL REGISTRATION: ClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in Patients with Autism”.
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spelling pubmed-37658332013-09-08 Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism Lv, Yong-Tao Zhang, Yun Liu, Min Qiuwaxi, Jia-na-ti Ashwood, Paul Cho, Sungho Charles Huan, Ying Ge, Ru-Cun Chen, Xing-Wang Wang, Zhao-Jing Kim, Byung-Jo Hu, Xiang J Transl Med Research BACKGROUND: Autism is a pervasive neurodevelopmental disorder. At present there are no defined mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem cell transplantation may offer a unique treatment strategy for autism due to immune and neural dysregulation observed in this disease. This non-randomized, open-label, single center phase I/II trial investigated the safety and efficacy of combined transplantation of human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) in treating children with autism. METHODS: 37 subjects diagnosed with autism were enrolled into this study and divided into three groups: CBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy), Combination group (9 subjects, received both CBMNC and UCMSC transplantation and rehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy). Transplantations included four stem cell infusions through intravenous and intrathecal injections once a week. Treatment safety was evaluated with laboratory examinations and clinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to assess the therapeutic efficacy at baseline (pre-treatment) and following treatment. RESULTS: There were no significant safety issues related to the treatment and no observed severe adverse effects. Statistically significant differences were shown on CARS, ABC scores and CGI evaluation in the two treatment groups compared to the control at 24 weeks post-treatment (p < 0.05). CONCLUSIONS: Transplantation of CBMNCs demonstrated efficacy compared to the control group; however, the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the CBMNC transplantation alone. There were no safety issues noted during infusion and the whole monitoring period. TRIAL REGISTRATION: ClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in Patients with Autism”. BioMed Central 2013-08-27 /pmc/articles/PMC3765833/ /pubmed/23978163 http://dx.doi.org/10.1186/1479-5876-11-196 Text en Copyright © 2013 Lv et al.; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/2.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Research
Lv, Yong-Tao
Zhang, Yun
Liu, Min
Qiuwaxi, Jia-na-ti
Ashwood, Paul
Cho, Sungho Charles
Huan, Ying
Ge, Ru-Cun
Chen, Xing-Wang
Wang, Zhao-Jing
Kim, Byung-Jo
Hu, Xiang
Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
title Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
title_full Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
title_fullStr Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
title_full_unstemmed Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
title_short Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
title_sort transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3765833/
https://www.ncbi.nlm.nih.gov/pubmed/23978163
http://dx.doi.org/10.1186/1479-5876-11-196
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