This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.

开始日期2023-08-28

申办/合作机构

University of Minnesota

100 项与 Mesenchymal stem cells(University of Minnesota) 相关的临床结果

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100 项与 Mesenchymal stem cells(University of Minnesota) 相关的专利（医药）

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项与 Mesenchymal stem cells(University of Minnesota) 相关的文献（医药）

2022-05-01·The Journal of Allergy and Clinical Immunology: In Practice1区 · 医学

Prospective Observational Study of Clinical Outcomes After Intravenous Magnesium for Moderate and Severe Acute Asthma Exacerbations in Children

1区 · 医学

Article

作者: Bacharier, Leonard B ; Gong, Wu ; Antoon, James W ; Akers, Wendell S ; Stewart, Thomas G ; Hartert, Tina V ; Arnold, Donald H ; Johnson, David P

BACKGROUNDThere is limited knowledge regarding whether intravenous magnesium (IV-Mg) improves outcomes in children with acute asthma exacerbations.OBJECTIVETo examine whether IV-Mg improves outcomes in children with moderate and severe exacerbations.METHODSWe performed a secondary analysis using data from a prospective observational cohort of children aged 5 to 17 years with moderate and severe exacerbations. Standardized treatment included systemic corticosteroid and inhaled albuterol, with consideration of IV-Mg (75 mg/kg) for patients with insufficient response after 20 minutes. Propensity score (PS) models were used to examine associations of IV-Mg treatment with change in the validated Acute Asthma Intensity Research Score, hospitalization rate, and time to spacing of inhaled albuterol of 4 hours or more among hospitalized participants.RESULTSAmong 301 children, median (interquartile range) age was 8.1 (6.4-10.2) years, 170 were Black (57%), 201 were male (67%), and 84 received IV-Mg (28%). In a PS covariate-adjusted multivariable linear regression model, IV-Mg treatment was associated with a 2-hour increase in the Acute Asthma Intensity Research Score (β-coefficient = 0.98; 95% confidence interval [CI], 0.20-1.77), indicating increased exacerbation severity. Three additional PS-based models yielded similar results. Participants receiving IV-Mg had 5.8-fold (95% CI, 2.8-11.9) and 6.8-fold (95% CI, 3.6-12.9) greater odds of hospitalization in PS-based multivariable regression models. Among hospitalized participants, there was no difference in time to albuterol of every 4 hours or more in a PS covariate-adjusted Cox proportional hazards model (hazard ratio = 1.2; 95% CI, 0.8-1.8).CONCLUSIONSAmong children with moderate and severe exacerbations, IV-Mg is associated with increased exacerbation severity, increased risk for hospitalization, and no acceleration in exacerbation resolution among hospitalized participants.

2022-03-03·Stem Cells Translational Medicine

Effects of Mesenchymal Stem Cell Transplantation on Cerebrospinal Fluid Biomarkers in Progressive Multiple Sclerosis

Article

作者: Ginzberg, Ariel ; Kassis, Ibrahim ; Petrou, Panayiota ; Karussis, Dimitrios ; Hallimi, Michelle

AbstractBackgroundNeurofilament light chains (NF-L) were shown to serve as a reliable biomarker of neurodegeneration in multiple sclerosis (MS). The chemokine receptor CXCL13 was shown to correlate with CNS inflammatory activity and to predict the future progression of MS.ObjectiveTo evaluate the levels of NF-L and CXCL13 in the cerebrospinal fluid (CSF) following treatment with mesenchymal stem cells (MSC) in patients with progressive MS.MethodsThe CSF samples were obtained from 48 patients with progressive MS who participated in a double-blind randomized phase II clinical trial that tested the effects of intrathecal (IT) or intravenous (IV) transplantation of mesenchymal stem cells (MSC), at baseline (before the first injection of the MSC) and at 6 months following treatment with MSC, or sham treatment. The CSF specimens were tested in a blinded way, using a single-molecule array (SIMOA) technique.FindingsThe CSF levels of NF-L were significantly lower at 6 months following treatment with MSC-IT when compared with the baseline, pre-treatment measurements (P = .026, Wilcoxon paired test). Nine out of 15 tested patients in the MSC-IT group had a reduction in NF-L levels of more than 50% (median decrease: −4449 pg/mL) when compared with 5/15 in the MSC-IV group (median decrease: −151 pg/mL) and 1/15 in the placebo group (median increase: +2450 pg/mL) (P = .001 for MSC-IT vs. placebo, chi-square test). CXCL13 levels were also reduced at 6 months following MSC-IT treatment but not to a statistically significant level.ConclusionsOur findings indicate possible neuroprotective effects of MSC transplantation in patients with MS.Clinical trial registrationNCT02166021

2020-12-01·Brain1区 · 医学

Beneficial effects of autologous mesenchymal stem cell transplantation in active progressive multiple sclerosis

1区 · 医学

Article

作者: Abramsky, Oded ; Hallimi, Michelle ; Paul, Friedemann ; Petrou, Panayiota ; Levy, Yarden ; Karussis, Dimitrios ; Oertel, Frederike Cosima ; Yaghmour, Nour ; Ginzberg, Ariel ; Hur, Tamir Ben ; Kassis, Ibrahim ; Backner, Yael ; Levin, Netta ; Scheel, Michael ; Benoliel, Tal

AbstractIn this study (trial registration: NCT02166021), we aimed to evaluate the optimal way of administration, the safety and the clinical efficacy of mesenchymal stem cell (MSC) transplantation in patients with active and progressive multiple sclerosis. Forty-eight patients (28 males and 20 females) with progressive multiple sclerosis (Expanded Disability Status Scale: 3.0–6.5, mean : 5.6 ± 0.8, mean age: 47.5 ± 12.3) and evidence of either clinical worsening or activity during the previous year, were enrolled (between 2015 and 2018). Patients were randomized into three groups and treated intrathecally (IT) or intravenously (IV) with autologous MSCs (1 × 106/kg) or sham injections. After 6 months, half of the patients from the MSC-IT and MSC-IV groups were retreated with MSCs, and the other half with sham injections. Patients initially assigned to sham treatment were divided into two subgroups and treated with either MSC-IT or MSC-IV. The study duration was 14 months. No serious treatment-related safety issues were detected. Significantly fewer patients experienced treatment failure in the MSC-IT and MSC-IV groups compared with those in the sham-treated group (6.7%, 9.7%, and 41.9%, respectively, P = 0.0003 and P = 0.0008). During the 1-year follow-up, 58.6% and 40.6% of patients treated with MSC-IT and MSC-IV, respectively, exhibited no evidence of disease activity compared with 9.7% in the sham-treated group (P < 0.0001 and P < 0.0048, respectively). MSC-IT transplantation induced additional benefits on the relapse rate, on the monthly changes of the T2 lesion load on MRI, and on the timed 25-foot walking test, 9-hole peg test, optical coherence tomography, functional MRI and cognitive tests. Treatment with MSCs was well-tolerated in progressive multiple sclerosis and induced short-term beneficial effects regarding the primary end points, especially in the patients with active disease. The intrathecal administration was more efficacious than the intravenous in several parameters of the disease. A phase III trial is warranted to confirm these findings.

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适应症	最高研发状态	国家/地区	公司	日期
肾上腺脑白质营养不良	临床1期	美国	University of Minnesota	2023-08-28

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ISC2020	N/A	急性缺血性卒中	777	IV-tPA	齋醖壓構窪鹽壓鑰觸齋(鹹衊簾製糧齋構範壓積) = 鑰鏇鹽繭範衊壓憲製顧願艱廠醖憲獵鑰醖壓築 (衊製壓遞獵遞鏇餘艱顧 ) 更多	-	2020-02-01
ISC2015	N/A	颅内出血	2,673	IV-tPA	選簾範窪獵壓簾鹹構遞(衊積範製窪艱簾鹽網壓) = 觸壓夢觸積製選鏇鏇壓壓構簾繭壓觸鏇糧鹽觸 (壓鹹顧鹹範獵選繭繭鹹 ) 更多	-	2015-02-01