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Table 2 Summary of the major clinical trials utilising MSCs as a treatment for T1D

From: CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: a path to clinical success?

Trial number Phase Intervention Outcomes Fresh/frozen Status Reference
NCT01068951 N/A Intravenous, autologous transplantation of MSCs (approximately 2 × 106 cells/kg body weight) Patients in the control arm showed losses in both C-peptide peak values and C-peptide when calculated as area under the curve during the first year. In MSC-treated patients, these responses were preserved or even increased. No side effects of MSC treatment were observed Fresh Completed 2014 [20]
NCT01374854 1/2 1 × 106/kg UC-MSCs are infused through the pancreatic artery along with BM-MNCs by interventional therapy and another same dose of UC-MSCs administered 1 week post-intervention C-peptide increased 105.7% in 20 of 21 responders versus 7.7% decrease in control subjects. HbA1C decreased 12.6% in treated versus 1.2% increase in control subjects. Daily insulin requirements decreased 29.2% in treated versus no change in control subjects. Fresh Completed 2012 [21]
NCT00703599 1/2 i.v. administration of autologous activated stromal vascular fraction derived from 100–120 ml lipoaspirates following mini-liposuction of abdominal adipose tissue Not reported Frozen Estimated completion of 2009 [22]
NCT01219465 1/2 i.v transfusion of UC-MSCs (2 × 107 cells/kg body weight) No reported acute or chronic side effects in MSC-treated versus saline control. Both HbA1c and C-peptide in MSC-treated patients were significantly better than either pre-therapy values or saline control patients during the follow-up period Fresh Completed 2012 [23]
NCT01996228 NCT01350219 1/2 Human UC-MSCs within the Stem Cell Educator device A single treatment provided lasting reversal of autoimmunity that allowed regeneration of islet β cells and improvement of metabolic control in subjects with long-standing T1D Fresh Recruiting [2427]
NCT02057211 2 Transfusion of autologous MSC versus sham MSC transfusion vs placebo control N/A Fresh Recruiting N/A
NCT01143168 1 Multiple transplantation of BM-MNC + UC-MSCs Not reported Frozen Estimated completion of 2011 Cellonis Biotech Pty Ltd.
NCT00646724 1/2 Co-transplantation of islet allograft and MSC autograft Not reported Frozen Estimated completion of 2012 N/A
NCT01322789 1/2 Four consecutive intravenous infusions, 1 week apart, followed by four consecutive infusions 1 month apart Not reported Frozen Estimated completion of 2015 N/A
NCT01496339 1/2 1 × 106/kg MenSCs are infused through the pancreatic artery or intravenously once a week in four consecutive therapies Not reported Frozen Estimated completion of 2014 S-Evans Biosciences Pty Ltd.
NCT02644759 1/2 Transplantation of autologous CD34+/CD133+ cells into the pancreatic artery and capillaries via interventional radiology techniques. Immunomodulation by incubation of autologous UC-MSCs for 3–6 h, and return of autologous WBCs back via intravenous injection N/A Fresh Ongoing, not recruiting Stem Cells Arabia
NCT00690066 2 Intravenous infusion of ex vivo cultured adult human MSCs vs placebo intravenous infusion of excipients of PROCHYMAL® Not reported Frozen Completed 2014 Osiris/Mesoblast International Sarl
NCT01157403 2/3 Intravenous autologous transplantation of BMSC (approximately 2.5 × 106 cells/kg body weight) Not reported Frozen Estimated completion of 2014 N/A
  1. Clinical trial data was acquired from using the search terms “mesenchymal stem cells” and “type 1 diabetes”
  2. BM-MNC bone marrow mononuclear cell, BMSC bone marrow-derived mesenchymal stem cell, CD cluster of differentiation, HbA1C glycosylated haemoglobin, i.v. intravenous, MenSC menstrual blood mesenchymal stem cell, MSC mesenchymal stem cell, N/A not available, T1D type 1 diabetes, UC-MSC umbilical cord mesenchymal stem cell, WBC white blood cell