Table 1 Overview of clinical trials using UC-MSCs in immune and inflammatory diseases
Indication | Clinical trial design | Cohort (UC-MSC/Control) | Posology | Aim | Results | Ref |
|---|---|---|---|---|---|---|
Type I diabetes mellitus | Randomized, double blind, controlled (placebo) | 29 (15/14) | 2.6 ± 1.2 × 107 Twice, 4W interval IV | Feasibility, safety, and efficacy | No acute or chronic side effects. HbA1c and C peptide improvement. | [29] |
Severe and refractory systemic lupus erythematosus | Single-arm | 16 | 1 × 106/kg IV | Safety and efficacy | No treatment-related deaths. Increase in peripheral T-reg cells. | [30] |
Refractory systemic lupus erythematosus | Two arms, controlled (glucocorticoids and cyclophosphamide) | 37 (17/20) | 3 × 107 IV | Safety and efficacy | No complications. Higher serum albumin and C3, lower anti-dsDNA. | [31] |
Multiple sclerosis | Randomized, controlled (AI and IS agents) | 23 (13/10) | 4 × 106/kg 3 times 2W interval IV | Efficacy | Decrease of Expanded Disability Status Scale (EDSS) scores and relapse occurrence. | [32] |
Multiple sclerosis | Phase I/phase II open-label, single-arm, single-center | 20 | 2 × 107/day 7 times IV | Feasibility, safety, and efficacy | No serious side effects. Inactive brain and cervical spinal cord lesions in 83% patients at 1 year. | [33] |
Rheumatoid arthritis | Phase I/II, single-arm, single-center | 64 | 2 × 107 IV | Long-term efficacy and safety | No serious side effects at 1 year and 3 years. Decrease of disease activity score at 1 year and 3 years. | [34] |
Crohn’s disease | Prospective, randomized, open- label, controlled (IS agents) | 82 (41/41) | 1 × 106/kg, 4 times, 1W interval IV | Safety and efficacy | No serious side effects. Decrease of Crohn’s disease activity index (CDAI), Harvey-Bradshaw index (HBI), and corticosteroid dosage. | [35] |
Chronic graft-versus-host disease (cGVHD) after hematopoietic stem-cell transplantation | Phase II, multicenter, randomized, double-blind, controlled (placebo) | 124 (62/62) | 3 × 107/month 4 times maximum IV | Safety and efficacy | Side effects not associated with MSC. Decrease of cGVHD cumulative incidence at 2 years. | [36] |
Acute liver allograft rejection | Randomized, controlled (IS agents) | 27 (14/13) | 1 × 106/kg 1–3 times, 4W interval IV | Feasibility and safety | No serious side effects. Decrease of ALAT level, improvement of allograft histology. | [37] |
Delayed graft function and acute rejection in deceased donor renal transplantation | Randomized, multicenter controlled | 42 (21/21) | 2 × 106/kg IV 5 × 106 renal arterial injection | Safety and feasibility | No serious side effects. Comparable graft and recipient survivals. | [38] |
Severe sepsis | Phase 1, single-center, open-label, dose-escalation | 15 | 1 × 106/kg 2 × 106/kg 3 × 106/kg IV | Safety and feasibility | No serious side effects up to 18-months of follow-up. | [39] |
HIV-1 infected immune non-responders | Pilot, randomized, open-label, controlled (HAART) | 13 (7/6) | 0.5 × 106/kg 3 times, 4W interval, IV | Safety and efficacy | Well tolerated. Increase of naive and central memory CD4 T-cell, restoration of IFN-γ and IL-2 production. | [40] |