Table 1

Summary of recent human cell therapy trials for stroke

Clinical trial/sponsorAgeTime after strokeAdditional selection criteriaCell typeRouteStroke locationPatients (n)Safety resultsEfficacy results
MASTERS/Athersys18–8324–48 hoursNIHSS 8–20, infarct 5-100cc, premorbid mRS 0–1Multistem adult-derived stem cell productIntravenousCortical129Similar 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, India18–757–29 daysNIHSS >7, GCS >8, BI <50, paretic arm or leg stable >48 hoursAutologous marrow-derived stem cellsIntravenous120
(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/Aldagen30–7513–19 daysNIHSS 7–22, mRS >3ALDHbr autologous marrow-derived stem cellsIntracarotid infusion distal to ophthalmicAnterior circulation ± subcortical29 IA, 19 sham12 SAE IA, 11 SAE sham; 0 cell-related SAE23 No difference in mRS, Barthel, NIHSS at 90 days or 1 year
PISCES-II/ReNeuron40–892–13 monthsParetic arm with NIHSS motor arm score 2–3CTX0E03 DP allogeneic human fetal neural stem cellsStereotaxic infusion into ipsilateral putamen21PendingPending
Sanbio18–756–60 monthsNIHSS>7, mRS 3–4, stable symptoms>3 weeksSB623 allogeneic marrow-derived stem cells transiently transfected with plasmid encoding Notch122 Stereotaxic infusion peri-infarctSubcortical ± cortical component24 1828 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 only6–60 monthsPersistent hemiparesis, Stable NIHSS over 4 weeks (Pt 2CTX0E03 DP allogeneic human neural stem cellsStereotaxic infusion into putamenSubcortical1116 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.