Table 2.
Clinical trials using mesenchymal stem cell-based therapy.
| Disease | Sample size |
Study Period |
MSC source |
Dosage | Administration Route |
Effects | Clinical Trial Stage |
Reference |
|---|---|---|---|---|---|---|---|---|
| GvHD | 55 Adults | 60 months | Allogeneic BM-MSCs | 0.4–9 × 106/ kg, 1–5 doses | I.V | CR (30/55) PR (9/55), NR (16/55) Increase overall survival in CR, no adverse events | Phase II | Le Blanc K et al, 2008[137] |
| GvHD | 31 Adults | 28 days | Allogeneic BM-MSCs | 2 or 8 × 106/ kg, 1 dose | I.V | CR (24/31), PR (5/31)NR (2/31), no adverse events. | Phase II | Kebriaei P et al, 2009[136] |
| GvHD | 2 Children | 18 months | UC-MSCs | 3.3 – 8.0 × 106/kg, 4 doses | I.V | CR (2), no adverse events. | Case Report | Wu KH et al, 2011[139] |
| GvHD | 13 Adults | 257 days | Allogeneic BM-MSC | 0.9–1.1 × 106/ kg, 2 doses | I.V | CR (2/13) PR (5/13), NR (6/13), no adverse events | Case series | Von Bonin M et al, 2009[138] |
| SLE | 40 Adults | 12 months | UC-MSCs | 1×106/kg, 2 dose | I.V | CR (13/40), P (11/40), NR (16/40), 7 recurrence, no adverse events. | Wang D et al, 2014[140] | |
| SLE | 35 Adults | 21 months | 8 receive BM-MSCs and 27 UC-MSCs | 1×106/kg/1–3 doses | I.V | CR (33/35), recurrence (2/35), Increase in Treg and decrease of Th17. No adverse events | Li X et al, 2013[145] | |
| SLE | 87 Adults | 27 months (mean) | BM-MSCs and UC-MSCs | 1×106/kg/ 1 dose | I.V | CR (43/87)at 4 years, P/NR (44/87), relapse 20/87at 4 years, no adverse events | Phase I/II | Wang D et al, 2013[141] |
| Multiple sclerosis and Amyotrophic lateral sclerosis | MS: 15 Adults ALS: 19 Adults | 6 months | Autologous BM-MSCs | MS: 6.32 × 107/kg ALS: 1.74 × 107. 1 dose | Intrathecal and I.V | CR (20/34), P/NR (14/30), no adverse events | Phase I/II | Karussis D et al, 2010[143] |
| Multiple sclerosis | 10 Adults | 10 months | Autogenous BM-MSCs | (1.1 – 2.0) × 106/kg. 1 dose | I.V | CR (10/10), improvement in visual acuity, visual evoked response latency and optic nerve area. No significant adverse events. | Phase IIA | Connick P et al, 2012[144] |
| Diabetes | 41 Adults | 24 months | Autologous BM-MSCs | Non state | I.M | Improved painless walking time, ankle-brachial index, transcutaneous oxygen pressure and magnetic resonance angiography. No serious adverse events | Phase I | Lu D et al, 2011[148] |
| Type 1 Diabetes | 29 Adolescents | 21 months | Allogeneic UC- MSCs | (1.5 – 3.2 × 107/kg | I.V | CR (3/15), P (9/15), NR (3/15) Improved recovery and regeneration of islet B-cells. No serious adverse events | Hu J. et al, 2013[146] | |
| Type 1 Diabetes | 11 | 23 months | Allogeneic ADMSCs | 4.6 × 107 −2.48 × 108 cells/dose (range) | I.V(Intraportal) | CR (11/11), Gradual decrease in insulin requirements and in Hb1Ac. No adverse events | Vanikar AV et al, 2010[147] | |
| Type 2 Diabetes | 10 Adults | 3 Months | Allogeneic placenta derived MSCs | 1.35 × 106/kg, 3 doses | I.V | CR (10/10) Decrease in insulin requirements. No adverse events | Phase I | Jiang R et al, 2011[149] |
| Type 2 Diabetes | 22 Adults | 12 months | UC-MSCs | 1 × 106/kg, 2 doses | I.V | CR (17/22) PR/NR (5/22) Improvement in B cell function, systemic inflammation (IL-6 and IL-1B) and T cells counts (CD3+ and CD4+). Adverse events (2/22) | Phase I/II | Liu X et al, 2014[150] |
| Idiopathic Pulmonary Fibrosis | 8 Adults | 6 months | Allogeneic Placenta derived MSCs | 1 × 106/kg or 2 × 106/kg | I.V | PR/NR (8/8). Small bowel obstruction, left lower lobe consolidation and mild episodes of bronchitis were reported as side effects (3/8) | Phase 1b | Chambers DC et al, 2013[151] |
| Idiopathic Pulmonary Fibrosis | 14 Adults | 12 months | Autologous AD-MSCs | 1.5 × 106/kg, 3 doses | Endobronchial | NR (14/14) Worsening cough and dyspnea were reported as adverse events (2/14) | Phase 1b | Tzouvelekis A et al, 2013[156] |
Abbreviations: GvHD: graft versus host disease; SLE: systemic lupus erythematosus; MSC: mesenchymal stem cells; UC-MSC: umbilical cord derived mesenchymal stem cell, BM-MSC: bone marrow derived mesenchymal stem cell; AD-MSC: adipose derived mesenchymal stem cell; I.V: intravenous; I.M: intramuscular; CR: complete response; PR: partial response; NR: no response.