Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke: Clinical Outcomes from a Phase I Safety Study.

Laskowitz, Daniel T

Bennett, Ellen R

Durham, Rebecca J

Volpi, John J

Wiese, Jonathan R

Frankel, Michael

Shpall, Elizabeth

Wilson, Jeffry M

Troy, Jesse

Kurtzberg, Joanne

2021-03-04T19:09:19Z

2021-03-04T19:09:19Z

2018-07

2021-03-04T19:09:18Z

Stroke is a major cause of death and long-term disability, affecting one in six people worldwide. The only currently available approved pharmacological treatment for ischemic stroke is tissue plasminogen activator; however, relatively few patients are eligible for this therapy. We hypothesized that intravenous (IV) infusion of banked unrelated allogeneic umbilical cord blood (UCB) would improve functional outcomes in patients with ischemic stroke. To investigate this, we conducted a phase I open-label trial to assess the safety and feasibility of a single IV infusion of non-human leukocyte antigen (HLA) matched, ABO matched, unrelated allogeneic UCB into adult stroke patients. Ten participants with acute middle cerebral artery ischemic stroke were enrolled. UCB units were matched for blood group antigens and race but not HLA, and infused 3-9 days post-stroke. The adverse event (AE) profile over a 12 month postinfusion period indicated that the treatment was well-tolerated in these stroke patients, with no serious AEs directly related to the study product. Study participants were also assessed using neurological and functional evaluations, including the modified Rankin Score (mRS) and National Institute of Health Stroke Scale (NIHSS). At 3 months post-treatment, all participants had improved by at least one grade in mRS (mean 2.8 ± 0.9) and by at least 4 points in NIHSS (mean 5.9 ± 1.4), relative to baseline. Together, these data suggest that a single i.v. dose of allogeneic non-HLA matched human UCB cells is safe in adults with ischemic stroke, and support the conduct of a randomized, placebo-controlled phase 2 study. Stem Cells Translational Medicine 2018;7:521-529.

2157-6564

2157-6580

https://hdl.handle.net/10161/22425

eng

Oxford University Press (OUP)

Stem cells translational medicine

10.1002/sctm.18-0008

Brain

Fetal Blood

Humans

Graft vs Host Disease

HLA Antigens

Magnetic Resonance Imaging

Treatment Outcome

Transplantation, Homologous

Aged

Middle Aged

Female

Male

Stroke

Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke: Clinical Outcomes from a Phase I Safety Study.

Journal article

Laskowitz, Daniel T|0000-0003-3430-8815

Troy, Jesse|0000-0001-5410-8146

Kurtzberg, Joanne|0000-0002-3370-0703

521

529

7

School of Medicine

Neurology, Neurocritical Care

Duke

Neurology

Clinical Science Departments

Duke Cancer Institute

Duke Innovation & Entrepreneurship

Pathology

Pediatrics, Transplant and Cellular Therapy

Institutes and Centers

Initiatives

Institutes and Provost's Academic Units

Pediatrics

Duke Clinical Research Institute

Neurobiology

Duke Science & Society

Duke Global Health Institute

Anesthesiology

Neurosurgery

Basic Science Departments

University Institutes and Centers

Biostatistics & Bioinformatics

Published

7