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. Author manuscript; available in PMC: 2017 Nov 2.
Published in final edited form as: J Stem Cell Regen Biol. 2017 Apr 10;3(2):10.15436/2471-0598.17.022. doi: 10.15436/2471-0598.17.022

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.