Allogeneic Stem Cell Therapy for Acute Ischemic Stroke: The Phase 2/3 TREASURE Randomized Clinical Trial

Area of Science:

• Regenerative Medicine
• Neuroscience
• Clinical Trials

Background:

• Cell therapy offers a promising avenue for treating stroke and other conditions.
• The efficacy of MultiStem (HLCM051), an allogeneic bone marrow-derived progenitor cell product, for ischemic stroke remains unestablished.

Purpose of the Study:

• To evaluate the safety and efficacy of MultiStem when administered to patients within 18 to 36 hours after ischemic stroke onset.

Main Methods:

• A multicenter, double-blind, placebo-controlled phase 2/3 randomized clinical trial (TREASURE) involving 206 patients with acute ischemic stroke.
• Patients received either intravenous MultiStem (1.2 billion cells) or placebo within 18 to 36 hours of stroke onset.
• Primary endpoints included safety and excellent functional outcome at 90 days, defined by a composite of modified Rankin Scale (mRS), NIHSS, and Barthel index scores.

Main Results:

• No significant differences were observed in the primary or secondary endpoints between the MultiStem and placebo groups.
• The proportion of patients achieving an excellent outcome at 90 days was similar: 11.5% for MultiStem versus 9.8% for placebo (P=.90).
• Adverse event frequencies were comparable between the treatment and placebo groups, indicating a similar safety profile.

Conclusions:

• Intravenous administration of MultiStem cell therapy within 18 to 36 hours of ischemic stroke onset is safe but did not demonstrate short-term efficacy.
• Exploratory analyses suggest further investigation may be warranted to identify specific patient subgroups who might benefit from MultiStem therapy.
• Additional research is required to fully determine the therapeutic potential of MultiStem for ischemic stroke treatment.