Table 1

Summary of recent human cell therapy trials for stroke

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	ALDH^br 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.