BackgroundAutism 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.Methods37 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.ResultsThere 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).ConclusionsTransplantation 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 registrationClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in Patients with Autism”.
ABSTRACT. We conducted a cohort study to investigate the role of 3 single-nucleotide polymorphisms of the excision repair crosscomplementation group 1 (ERCC1) gene on the response to chemotherapy and clinical outcomes of non-small cell lung cancer (NSCLC). A total of 163 patients with newly diagnosed and histopathologically confirmed primary NSCLC were examined in our study and were followed up until December 2012. ERCC1 rs11615, rs3212986, and rs2298881 were selected and genotyped. Of the 163 patients, 86 patients showed a complete response and partial response to chemotherapy (52.76%), while 91 patients (55.83%) died from NSCLC during the follow-up period with a median survival time of 19.3 months (range, 2-60 months). Multivariate regression analysis showed that individuals carrying the rs11615 TT genotype and T allele had a significantly lower response rate to chemotherapy using the rs11615 CC genotype as the reference. For rs3212986, carriers of the rs3212986 AA genotype and A allele had a significantly lower response rate to chemotherapy when compared with the CC genotype. In the Cox proportional hazards model, patients carrying the rs11615 TT genotype and T allele and the rs3212986 AA genotype and A allele were significantly associated with increased risk of death from NSCLC. We found that polymorphisms in ERCC1 rs11615 and rs3212986 were associated with poor response to chemotherapy and shorter survival time of advanced NSCLC.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.