Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial

INTRODUCTION: We conducted a phase 1 clinical trial in nine patients with mild-to-moderate Alzheimer's disease to evaluate the safety and dose-limiting toxicity of stereotactic brain injection of human umbilical cord blood–derived mesenchymal stem cells (hUCB-MSCs). METHODS: The low- (n = 3) and high-dose (n = 6) groups received a total of 3.0 × 10(6) cells/60 μL and 6.0 × 10(6) cells/60 μL, respectively, into the bilateral hippocampi and right precuneus. RESULTS: No patient showed serious adverse events including fever during the 24-month follow-up period. During the 12-week follow-up period, the most common acute adverse event was wound pain from the surgical procedure (n = 9), followed by headache (n = 4), dizziness (n = 3), and postoperative delirium (n = 3). There was no dose-limiting toxicity. DISCUSSION: Administration of hUCB-MSCs into the hippocampus and precuneus by stereotactic injection was feasible, safe, and well tolerated. Further trials are warranted to test the efficacy. CLINICAL TRIAL REGISTRATION: ClinicalTrial.gov identifier NCT01297218 and NCT01696591.