Clinical trial/sponsor | Age | Time after stroke | Additional selection criteria | Cell type | Route | Stroke location | Patients (n) | Safety results | Efficacy results |
MASTERS/Athersys | 18–83 | 24–48 hours | NIHSS 8–20, infarct 5-100cc, premorbid mRS 0–1 | Multistem adult-derived stem cell product | Intravenous | Cortical | 129 | Similar SAE at 1 year 22(34%) versus 24 (39%) placebo, Lower mortality—5 deaths (8%) versus 9deaths (15%) in placebo19 | No effect on 90-day Global Stroke Recovery Assessment (mRS 0–2, NIHSS increase by 75%, Barthel Index >95) but trend towards improved outcome with earlier delivery of cells19 |
InveST/Department of Biotechnology, India | 18–75 | 7–29 days | NIHSS >7, GCS >8, BI <50, paretic arm or leg stable >48 hours | Autologous marrow-derived stem cells | Intravenous | 120 (58 cell therapy) | 61 AE (33%) and eight deaths versus 60 AEs (36%) and five deaths placebo22 | No effect on 180-day Barthel Index Score, mRS shift or score >3, NIHSS, change of infarct volume22 | |
RECOVER-Stroke/Aldagen | 30–75 | 13–19 days | NIHSS 7–22, mRS >3 | ALDHbr autologous marrow-derived stem cells | Intracarotid infusion distal to ophthalmic | Anterior circulation ± subcortical | 29 IA, 19 sham | 12 SAE IA, 11 SAE sham; 0 cell-related SAE23 | No difference in mRS, Barthel, NIHSS at 90 days or 1 year |
PISCES-II/ReNeuron | 40–89 | 2–13 months | Paretic arm with NIHSS motor arm score 2–3 | CTX0E03 DP allogeneic human fetal neural stem cells | Stereotaxic infusion into ipsilateral putamen | 21 | Pending | Pending | |
Sanbio | 18–75 | 6–60 months | NIHSS>7, mRS 3–4, stable symptoms>3 weeks | SB623 allogeneic marrow-derived stem cells transiently transfected with plasmid encoding Notch122 | Stereotaxic infusion peri-infarct | Subcortical ± cortical component24 | 18 | 28 SAE, 0 cell-related SAE25 | Improved ESS at 6 months (p<0.01) and 12 months (p<0.001) Improved NIHSS at 6 months (p<0.01) and 12 months(p<0.001) Improved Fugl-Meyer at 6 months (p<0.001) and 12 months(p<0.001)25 |
PISCES/ReNeuron | >60, male only | 6–60 months | Persistent hemiparesis, Stable NIHSS over 4 weeks (Pt 2 | CTX0E03 DP allogeneic human neural stem cells | Stereotaxic infusion into putamen | Subcortical | 11 | 16 SAE (in nine patients), 0 cell-related SAE28 | Improved NIHSS at 2 years (p=0.002), No change, Barthel Index, MMSE, Ashworth, mRS28 29 |
AE, Adverse Event; ARAT, Action Research Arm Test; BI, Barthel Index; DP, drug product; ESS, European Stroke Scale; IA, intra-arterially; MASTERS, Multistem Administration for Stroke Treatment and Enhanced Recovery Study; MMSE, Mini-Mental Status Examination; mRS, modified Rankin Score; NIHSS, National Institutes of Health Stroke Scale; PISCES, Pilot Investigation of Stem Cells in Stroke; SAE, Serious aAverse Events.