Intrathecal versus intravenous umbilical cord mesenchymal stem cells for ischemic stroke sequelae

Stroke is a leading cause of death worldwide. Traditional treatments have limitations, stem cell therapy has potential for regeneration after ischemic stroke. This study evaluated the safety and efficacy of allogeneic umbilical cord-derived mesenchymal stem cell (UC-MSC) infusion via the intravenous (IV) and intrathecal (IT) routes for treating neurological sequelae after ischemic stroke.

This phase II randomized controlled trial involved 32 patients aged 40-75 years with neurological sequelae after ischemic stroke. The patients were randomly assigned into two groups: 16 received two IT UC-MSC infusions plus rehabilitation therapy, and 16 received two IV UC-MSC infusions plus rehabilitation therapy. Additionally, 16 matched controls, paired with the IT group by sex, age (±5 years), and NIHSS, received only rehabilitation. UC-MSCs were administered at 1.5 × 106 cells/kg at baseline and 3 months. Outcomes were assessed at baseline, 3, 6, and 12 months using NIHSS, FIM, MAS, FMS, and SF-36.

No severe adverse events related to UC-MSC therapy were observed. Adverse event rate was lower in the IV group than the IT group. At 6 months, the IV group demonstrated significant improvements in NIHSS (p = 0.046), FIM (p = 0.028), and SF-36 (p < 0.001). At 12 months, both UC-MSC groups showed significant improvements, with greater effects in the IV group (p < 0.001 for SF-36).

Both IV and IT UC-MSC infusions improved neurological recovery and quality of life, with fewer adverse events in the IT group.

NCT05292625.