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. Author manuscript; available in PMC: 2013 Sep 2.
Published in final edited form as: Annu Rev Biomed Eng. 2010 Aug 15;12:87–117. doi: 10.1146/annurev-bioeng-070909-105309

Table 1.

Examples of clinical trials of mesenchymal stem cell therapy reported in the academic literature

Indication Enrollment (Ntotal; Ncontrol, Ncells) Trial design Cell mass MOA Measured parameters and follow-up time after CTx Study conclusions Reference
BMTx (23; 15, 8) Local advanced or metastatic breast cancer with high-dose chemotherapy and PBPC Tx given CTx at 1 or 24 h later 1.5–3.9 × 106 CD34+ cells kg−1 and 2.2 × 106 MSCs kg−1 Neutrophil and platelet counts (daily)
CFU (42 days)
Mortality (100 days)

  1. Prompt hematopoietic engraftment of 8 days

  2. 70% CFU recovery

  3. One patient death after 100 days

 35
OI (7; 2, 5) Diagnosed severe type III OI with similar growth rates 5.7–7.5 × 106 bone marrow cells kg−1 iv Growth analysis (6 months) Increased growth rates that slowed over time 38
MLD (5; 1, 4) Patients with successfully matched bone marrow transplants 2–10 × 106 MSCs kg−1 iv Nerve conduction velocity
Mental/physical exams

  1. No toxicity

  2. Improved nerve conduction velocity

  3. No clinical change in overall mental or physical status

 39
ALS (7; 5, 2) Diagnosed ALS with severe lower-limb and mild upper-limb impairment MSCs suspended in CSF infused into T7–T9 exposed spinal cord MRI (3, 6 months)
Neuromuscular exam (3 months)

  1. No adverse reactions

  2. No structural MRI changes

  3. Mild increase in muscle strength of a lower-limb muscle group

 40
MI (69; 35, 34) CTx 10 days after PCI 48–60 × 109 MSCs into coronary artery lesion Echocardiography (monthly)
PET (3, 6 months)
EKG (3 months)

  1. Decreased akinetic, dyskinetic, hypokinetic segments

  2. Increased wall movement at infract

  3. LVEF higher

 9
GvHD (56; 10, 46) Open-label, multicenter trial of hematological cancer patients treated with CTx 4 days prior to haploidentical BMTx 1, 5, or 10 × 106 MSCs kg−1 iv Hematopoietic recovery (daily)
Acute GvHD

  1. Prompt hematopoietic engraftment in most patients

  2. 23 of 46 patients did not undergo acute GvHD, with 11 showing longer relapse time

 12

Abbreviations: ALS, amyotrophic lateral sclerosis; BMTx, bone marrow transplant; CFU, colony forming unit; CSF, cerebrospinal fluid; CTx, cell transplant; EKG, electrocardiogram; GvHD, graft-versus-host disease; iv, intravenous; LVEF, left ventricular ejection fraction; MI, myocardial infarction; MLD, metachromatic leukodystrophy; MOA, method of administration; MRI, magnetic resonance imaging; MSC, mesenchymal stem cell; OI, osteogenesis imperfecta; PBPC, peripheral blood progenitor cell; PCI, percutaneous coronary intervention; PET, positron emission tomography; Tx, transplantation.