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Positive Trial Results Supports Umbilical Cord Blood Therapy for the Treatment of Stroke

Review of “Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke: Clinical Outcomes from a Phase 1 Safety Study” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Current statistics suggest that someone in the United States suffers a stroke every 40 seconds and, given that stroke prevalence increases with advancing age in both males and females [1], stroke will likely remain as a significant cause of death and long-term disability in the coming future. Cell-based therapies aim to improve recovery following ischemic stroke, caused by a lack of blood flow to the brain, via the modulation of the immune system, the secretion of neurogenic and angiogenic factors, and the reduction of infarct volume. 

Umbilical cord blood (UCB) represents a safe, readily-available, cryopreserved, banked and immunologically tolerant stem cell-rich product that does not require the invasive collection of autologous cells via bone marrow harvesting or peripheral stem cell collection from already fragile patients [2]. Now, new research led by Ellen R. Bennett (Duke University, Durham, North Carolina, USA) sought to determine the potential for UCB therapy for the treatment of stroke [3].

Laskowitz et al. assessed the consequences of a single intravenous infusion of non‐human leukocyte antigen-matched, blood type matched, unrelated allogeneic UCB in ten participants (male adults 18–90 years of age, median age of 65.5 years, 3 to 9 days following acute middle cerebral artery ischemic stroke. Encouragingly, the study reported no serious adverse effects over a 12-month post-infusion period, indicating safety, while neurological and functional evaluations provided evidence of significant improvements by three months post-infusion relative to baseline. At this point, magnetic resonance imaging revealed no significant increase in infarct volume, unexpected bleeding, or other safety concerns when compared to baseline scans.

While the results suggest that intravenous infusion of UCB represents a non-invasive, safe, and feasible means to treat acute ischemic stroke, the authors do note limitations, which include the small sample size and the open‐label, non-randomized design. For these reasons, they have initiated a larger phase 2 randomized, placebo‐controlled, double‐blind trial to evaluate umbilical cord blood therapy for the treatment of stroke.

To hear the results from new studies of UCB therapy, stay tuned to the Stem Cells Portal!


  1. Benjamin EJ, Virani SS, Callaway CW, et al., Heart Disease and Stroke Statistics—2018 Update: A Report From the American Heart Association. Circulation 2018;137:e67-e492.
  2. Kim Y-J and Broxmeyer HE, Immune regulatory cells in umbilical cord blood and their potential roles in transplantation tolerance. Critical Reviews in Oncology / Hematology 2011;79:112-126.
  3. Laskowitz DT, Bennett ER, Durham RJ, et al., Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke: Clinical Outcomes from a Phase I Safety Study. STEM CELLS Translational Medicine 2018;7:521-529.