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Table 1 Clinical Studies Utilizing MSCs in the Treatment of Skeletal Disease

From: Mesenchymal stem cells (MSCs) as skeletal therapeutics–an update

References Sample size Cell type Delivery route Treatment outcomes
Osteogenesis Imperfecta
Horwitz et al., 2001 [67] 3 5.5-6.2 × 108 cells per Kg, Allogeneic bone marrow stromal cells (MSCs) Transplantation Total body bone mineral content improved from 45 % to 77 % above baseline. Growth velocity improved. The rate of fractures reduced as documented by radiographs.
Horwitz et al., 2002 [68] 6 1-5 × 106 cells per Kg, MSCs transduced with retroviruses Intravenous infusions, two doses with 8–21 days apart Patients experienced significant improvement in growth velocity without concomitant increase in bone mineral content
Le Blanc et al., 2005 [69] Prenatal female fetus 6.5 × 106, HLA mismatched fetal MSCs Intra-uterine injection X-ray absorptiometry showed 48 % skeletal mineralization compared to age matched counterpart
Götherström et al., 2014 [70] Female fetus with type IV OI 30 × 106 cells per kg followed by postnatal dose of 10 × 106, Human fetal MSCs Intrauterine implantation at 31 weeks of gestation. Thereafter, i.v infusion at 13 month age Patient was followed for her normal growth trajectory with no alloreactivity from received MSCs
Infantile Hypophosphatasia
Whyte et al., 2003 [75] 8 mo old girl 2.1 × 106 followed by SCB of 2.92 × 107 mononuclear cells per Kg recipient weight, Haplo-identical marrow stromal cells Bone marrow transplantation Striking improvements were seen in skeletal mineralization soon after SCB
Cahill et al., 2007 [72] 8 mo old girl Four bone fragments (2 mm × 10 mm) + MSCs Two fragments intraperitonealy and two subcutaneously Bone mineral contents were increased upto 46 % revealed by x-ray absorptiometry. No change in serum alkaline phosphatase levels was observed
Stenderup et al., 2001 [76] 13 1 × 105 cells per cm2, MSCs from Bone marrow aspirate - Bone remodeling and turnover occurred at faster rate in osteoporotic patients. However, it was dependent on the continuous availability of osteoprogenitor cells, growth factors and hormones.
Wakitani et al., 2002 [91] 12 1.3 × 107, Autologous MSCs Surgical implantation No significant improvement was seen on clinical evaluation, Histological examination revealed hyaline like cartilage
Centeno et al., 2008 [93] 1 22.4 × 106, MSCs in PBS + PL + dexamethasone Percutaneous injection MRI showed improvement in volume of meniscus and cartilage
Pak, 2011 [92] 2 8.3 cm3, mixture of Autologous ADSCs, PRP, dexamethasone, hyaluronic acid Intra-articular injection At 12 week, significant improvement in pain (more than 90 %) and flexion of knee was experienced by patients. MRI revealed improved cartilage thickness
Davatchi et al., 2011 [94] 4 8-9 × 106, Autologous MSCs Intra-articular injection Mild improvement in subjective and objective symptoms was observed.
Orozco et al., 2013 [95] 12 40 × 106, Autologous MSCs Intra-articular injection Algofunctional indices strongly indicated clinical efficacy of injected MSCs. T2 mapping demonstrated significantly improved cartilage quality in 11 out of 12 patients.
Jo et al., 2014 [96] 18 1.0 × 108 Adipose tissue derived MSCs Intra-articular Injection 6 months follow up showed reduction in WOMAC score, MRI findings revealed reduction in the size of cartilage defect
Vega et al., 2015 [97] 15 40 × 106 allogenic MSCs Intra-articular Injection Significant improvements in algofunctional indices versus controls and improvements in the quality of cartilage as assessed by T2 measurements
  1. MSCs mesenchymal stem cells, PBS phosphate buffered saline, MRI magnetic resonance imaging, SCB stromal cell boost, HLA human leukocyte antigen, PRP platelet rich plasma WOMAC Western Ontario and McMaster Universities Arthritis Index