Effect of umbilical cord mesenchymal stromal cells on motor functions of identical twins with cerebral palsy: pilot study on the correlation of efficacy and hereditary factors

Wang, Xiaodong; Hu, Hezhen; Hua, Rongrong; Yang, Jing; Pei, Zhihua; Niu, Xinxin; Cheng, Hao‐Tsai; Dai, Guohao; Liu, Xuebin; Zhang, Zan; An, Yu

doi:10.1016/j.jcyt.2014.09.010

Cited by 52 publications

(28 citation statements)

References 23 publications

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“…Promising safety and efficacy data were presented in several case reports regarding hUC-MSCs concomitant with [30] or without [31,32] rehabilitation. However, few randomized trials focused on hUC-MSC transplantation intravenously concomitant with rehabilitation.…”

Section: Discussionmentioning

confidence: 99%

Therapeutic evidence of umbilical cord-derived mesenchymal stem cell transplantation for cerebral palsy: a randomized, controlled trial

Huang

Che

et al. 2020

Stem Cell Res Ther

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Background: Cerebral palsy (CP) is a syndrome of childhood movement and posture disorders. Clinical evidence is still limited and sometimes inconclusive about the benefits of human umbilical cord mesenchymal stem cells (hUC-MSCs) for CP. We conducted a randomized trial to evaluate the safety and efficacy of hUC-MSC transplantation concomitant with rehabilitation in patients with CP. Methods: Eligible patients were allocated into the hUC-MSC group and control group. In addition to rehabilitation, the patients in the hUC-MSC group received four transfusions of hUC-MSCs intravenously, while the control group received a placebo. Adverse events (AEs) were collected for safety evaluation in the 12-month follow-up phase. Primary endpoints were assessed as activities of daily living (ADL), comprehensive function assessment (CFA), and gross motor function measure (GMFM) scales. In addition, cerebral metabolic activity was detected by 18 F-FDG-PET/ CT to explore the possible mechanism of the therapeutic effects. Primary endpoint data were analyzed by ANOVA using SPSS version 20.0. Results: Forty patients were enrolled, and 1 patient withdrew informed consent. Therefore, 39 patients received treatments and completed the scheduled assessments. No significant difference was shown between the 2 groups in AE incidence. Additionally, significant improvements in ADL, CFA, and GMFM were observed in the hUC-MSC group compared with the control group. In addition, the standard uptake value of 18 F-FDG was markedly increased in 3 out of 5 patients from the hUC-MSC group at 12 months after transplantation. Conclusions: Our clinical data showed that hUC-MSC transplantation was safe and effective at improving the gross motor and comprehensive function of children with CP when combined with rehabilitation. Recovery of cerebral metabolic activity might play an essential role in the improvements in brain function in patients with CP. The therapeutic window, transfusion route, and dosage in our study were considerable for reference in clinical application. Trial registration: Chictr.org.cn, ChiCTR1800016554. Registered 08 June 2018-retrospectively registered. The public title was "Randomized trial of umbilical cord-derived mesenchymal stem cells for cerebral palsy."

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Section: Discussionmentioning

confidence: 99%

Therapeutic evidence of umbilical cord-derived mesenchymal stem cell transplantation for cerebral palsy: a randomized, controlled trial

Huang

Che

et al. 2020

Stem Cell Res Ther

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“…Intrathecal MSC delivery has been evaluated for cerebral palsy and autism spectrum disorders and is technically feasible in most children, including premature neonates. Wang et al reported that allogeneic UCB‐MSCs given intrathecally to eight pairs of identical twins with cerebral palsy resulted in improvements in gross motor function . All patients received four intrathecal infusions of 1–1.5 × 10 7 cells 3–5 days apart.…”

Section: Clinical Studies Of Msc Therapymentioning

confidence: 99%

“…There may be underlying genetic factors dictating response to MSC therapy. Initial investigations toward elucidating these factors were conducted in a trial of UCB‐MSCs for cerebral palsy in twins . Significant improvements in gross motor function were observed, and these improvements were highly correlated between individuals of an identical twin pair, but not between twin pairs.…”

Section: Clinical Studies Of Msc Therapymentioning

confidence: 99%

Concise Review: Mesenchymal Stem Cell Therapy for Pediatric Disease: Perspectives on Success and Potential Improvements

Nitkin

Bonfield

2016

Stem Cells Translational Medicine

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Mesenchymal stem cells (MSCs) represent a potentially revolutionary therapy for a wide variety of pediatric diseases, but the optimal cell‐based therapeutics for such diversity have not yet been specified. The published clinical trials for pediatric pulmonary, cardiac, orthopedic, endocrine, neurologic, and hematologic diseases provide evidence that MSCs are indeed efficacious, but the significant heterogeneity in therapeutic approaches between studies raises new questions. The purpose of this review is to stimulate new preclinical and clinical trials to investigate these factors. First, we discuss recent clinical trials for pediatric diseases studying MSCs obtained from bone marrow, umbilical cord and umbilical cord blood, placenta, amniotic fluid, and adipose tissue. We then identify factors, some unique to pediatrics, which must be examined to optimize therapeutic efficacy, including route of administration, dose, timing of administration, the role of ex vivo differentiation, cell culture techniques, donor factors, host factors, and the immunologic implications of allogeneic therapy. Finally, we discuss some of the practicalities of bringing cell‐based therapy into the clinic, including regulatory and manufacturing considerations. The aim of this review is to inform future studies seeking to maximize therapeutic efficacy for each disease and for each patient. Stem Cells Translational Medicine 2017;6:539–565

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“…Stem cell therapy for cerebral palsy was shown to be safe and effective in human studies as well [11, 16–23]. Nevertheless, the number of studies on this promising intervention are still limited.…”

Section: Introductionmentioning

confidence: 99%

Outcomes of autologous bone marrow mononuclear cells for cerebral palsy: an open label uncontrolled clinical trial

Nguyen

Nguyễn

et al. 2017

BMC Pediatr

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BackgroundStem cell therapy has emerged as a promising method for improving motor function of patients with cerebral palsy. The aim of this study is to assess the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation in patients with cerebral palsy related to oxygen deprivation.MethodsAn open label uncontrolled clinical trial was carried out at Vinmec International Hospital. The intervention consisted of two administrations of stem cells, the first at baseline and the second 3 months later. Improvement was monitored at 3 months and 6 months after the first administration of stem cells, using the Gross Motor Function Measure (GMFM) and Modified Ashworth Score which measures muscle tone.ResultsNo severe complications were recorded during the study. After transplantation, 12 patients encountered fever without infections and 9 patients experienced vomiting which was easily managed with medications. Gross motor function was markedly improved 3 months or 6 months after stem cell transplantation than at baseline. The post-transplantation GMFM-88 total score, each of its domains and the GMFM-66 percentile were all significantly higher (p-value < 0.001). Muscle spasticity also reduced significantly after transplantation (p-value < 0.001). The therapy was equally effective regardless of sex, age and GMFCS level (p-value > 0.05).ConclusionAutologous bone marrow mononuclear cell transplantation appears to be a safe and effective therapy for patients with cerebral palsy.Trial registration ClinicalTrials.gov Identifier: NCT02569775. Retrospectively registered on October 15, 2015.Electronic supplementary materialThe online version of this article (doi:10.1186/s12887-017-0859-z) contains supplementary material, which is available to authorized users.

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Effect of umbilical cord mesenchymal stromal cells on motor functions of identical twins with cerebral palsy: pilot study on the correlation of efficacy and hereditary factors

Cited by 52 publications

References 23 publications

Therapeutic evidence of umbilical cord-derived mesenchymal stem cell transplantation for cerebral palsy: a randomized, controlled trial

Therapeutic evidence of umbilical cord-derived mesenchymal stem cell transplantation for cerebral palsy: a randomized, controlled trial

Concise Review: Mesenchymal Stem Cell Therapy for Pediatric Disease: Perspectives on Success and Potential Improvements

Outcomes of autologous bone marrow mononuclear cells for cerebral palsy: an open label uncontrolled clinical trial

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