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Allogeneic stem cell therapy for ischemic stroke is safe, but is it effective?
Intravenous allogeneic stem cell therapy within 18 to 36 hours of ischemic stroke onset was safe but did not improve short-term outcomes compared with placebo, according to study results from a phase 2/3 trial published in JAMA Neurology.
“However, efficacy was suggested in patients with large infarcts and possibly in younger patients based on exploratory subgroup analyses with no correction for multiple comparisons,” wrote corresponding study author Toshiya Osanai, MD, of the Hokkaido University Department of Neurosurgery in Japan, and study coauthors.
The double-blind, randomized clinical trial investigated the efficacy and safety of MultiStem, a bone marrow–derived, allogeneic, multipotent adult progenitor cell product, administered within 18 to 36 hours of ischemic stroke onset. More than 44 academic and clinical centers in Japan participated in the trial, which included a total 206 patients. Among them, 104 received intravenous MultiStem, and 102 received intravenous placebo.
There was no significant difference between treatment groups for the primary endpoint of excellent outcome at day 90, which was a composite of a modified Rankin Scale (mRS) score of 1 or less, a National Institutes of Health Stroke Scale score of 1 or less, and a Barthel index score of 95 or higher. Rates of excellent outcomes at day 90 were 11.5% in the MultiStem group and 9.8% in the placebo group, according to the study.
Similarly, the trial showed no between-group differences in secondary endpoints, which included excellent outcome at day 365, mRS score distribution at days 90 and 365, and mRS score of 0 to 1 and 0 to 2 at day 90.
The frequency of adverse events, too, was similar between treatment groups. No grade 3 or 4 allergic reactions occurred.
In exploratory subgroup analyses that looked at mRS scores of 2 or less at day 90 with no correction for multiple comparisons, patients with an ischemic core volume of 50 mL or less had significantly better outcomes with MultiStem compared with placebo (29.6% vs 8.1%), researchers reported. Patients aged 64 years and younger also tended to have better outcomes with MultiStem.
“The results of this study support the safety of MultiStem, but further research is needed to determine whether MultiStem therapy for ischemic stroke has a beneficial effect in patients who meet specific criteria,” the authors wrote.
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