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Table 2 Recent clinical use of MSCsa in phase I/II trials

From: Mesenchymal stem cells: from experiment to clinic

Patients, number

Source of MSCs

Phenotype

Culture

Cells infused

Dosage

Adverse effects

MSC survival

Follow-up

Outcome

5-year patient survival

Reference

Stroke, 16

Autologous iliac crest

> 90% SH2

DMEM, 10% FCS, Cryo

5 × 107 twice

2 doses, 2-week interval

None

ND

To 5 years

HR for MSCs = 0.344

Con 34%

MSCs 72%

[114]

MI, 10

Autologous iliac crest

CD73+ CD90+ CD105+

DMEM, 10%FCS

7.5 × 106 MSCs + EPCs

1 dose

None

ND

6 months

LVEF 12% up

--

[115]

MI, 53

Allogeneic unmatched 'prochymal'

CD105+ CD166+ CD45-

Cryob

0.5 to 5 × 106/kg

1 dose, intravenous

MSCs 5 pt Placebo 7

ND

6 months

FEV1 up, LVEF up, arrhythmia down

--

[126]

ALS, 10

Autologous iliac crest

CD29+ CD44+ CD90+ CD105+ CD166+

Cambrex

MSC medium 10% FCS

11.4 to 120 × 106

1 dose, thoracic spine

None

ND

4 years

MSCs tolerated, SC scars

--

[116]

ALS, 19 MS, 15

Autologous iliac crest

CD29+ CD73+ CD90+ CD105+ CD166+

DMEM, 10% FCS

ALS 5 × 107 MS 6 × 107 +/- ferumoxide

1 dose, intrathecal; 1 dose, intravenous

None; CD4/25 Treg cells up

Possible

6-25 months

ALSFRS stable, EDSS better

--

[32]

Refractory Crohn's Disease, 10

Autologous iliac crest

CD73+ CD90+ CD105+

DMEM, 10% FCS, Cryo

1 to 2 × 106/kg [mean wt 57 kg, range 46-113 kg

2 doses, 1-week interval

Headache (3 patients) Allergy (1 patient)

ND

14 weeks

CDAI fall (5 patients; > 70 in 3 patients);3 patients worse

--

[117]

Paediatric acute leukaemia 8

Haploid parent BM

Adherent CD phenotype ND

Cryob

6 × 104 to 107/kg MSCs + UCBT

1 or 2 doses, 3-week interval

None

None

6.8 years, no chronic GVHD

All patients PMN+ at 9 to 28 days

63%

[109]

Leukaemia 12c

HLA match opposite gender HCT

CD44+ CD73+ CD90+ CD103+

ND

ND

1 dose

ND

None

0.9-138 months

HCT success, no MSCs took

ND

[110]

  1. aAbbreviations: ALS, amyotrophic lateral sclerosis; ALSFRS, ALS Functional Rating Scale; BM, bone marrow; CDAI, Crohn's disease activity index; Con, control; Cryo, cryogenic mesenchymal stem-cell preservation using dimethylsulphoxide; DMEM, Dulbecco's modified Eagle medium; EDSS, Expanded Disability Status Scale; EPC, endothelial precursor cell; FCS, foetal calf serum; FEV1, forced expiratory volume in 1 second; GVHD, graft-versus-host disease; HCT, haematopoietic cell transplant. HR, hazard ratio; LVEF, left ventricular ejection fraction; ND, not determined; PMN, polymorphonuclear leukocyte (surrogate for HSC engraftment); SC, spinal cord; UCBT, umbilical-cord blood transplant (unrelated).
  2. bOsiris Therapeutics.
  3. cNote this study did not infuse MSCs, but analysed cultured recipient BM MSCs after sex-mismatched BM transplant for presence of donor MSCs.