Related Experiment Video
Updated: Apr 7, 2026
Delayed Intramyocardial Delivery of Stem Cells after Ischemia Reperfusion Injury in a Murine Model
Published on: September 3, 2020
A Phase II Dose-Escalation Study of Allogeneic Mesenchymal Precursor Cells in Patients With Ischemic or Nonischemic
Emerson C Perin1, Kenneth M Borow2, Guilherme V Silva2
1From the Stem Cell Center (E.C.P., G.V.S., Y.Z., J.T.W.), Adult Cardiology (E.C.P., G.V.S., J.T.W.), Texas Heart Institute, Houston; Borow Consulting Group, LLC, Bryn Mawr, PA (K.M.B.); Division of Cardiology, School of Medicine, University of California San Diego (A.N.D.M.); Heart and Vascular Institute, Center for Heart and Vascular Quality, Outcomes, and Clinical Research, University of Pittsburgh Medical Center, PA (O.C.M.); Heart and Vascular Institute, Swedish Medical Center, Seattle, WA (P.P.H.); Minneapolis Heart Institute Foundation, Abbott Northwestern Hospital, MN (J.H.T.); Monash University, Melbourne, Australia (H.K.); Mesoblast, Inc, New York (D.S.); Mesoblast, Inc, Melbourne, Victoria, Australia (S.I.); and Division of Cardiology, Cedars-Sinai Heart Institute, Los Angeles, CA (T.D.H.). eperin@texasheart.org.
Rationale:
Allogeneic mesenchymal precursor cells (MPCs) have been effective in large animal models of ischemic and nonischemic heart failure (HF).
Objective:
To evaluate the feasibility and safety of 3 doses (25, 75, or 150 million cells) of immunoselected allogeneic MPCs in chronic HF patients in a phase 2 trial.
Methods And Results:
We sequentially allocated 60 patients to a dosing cohort (20 per dose group) and randomized them to transendocardial MPC injections (n=15) or mock procedures (n=5). The primary objective was safety, including antibody testing. Secondary efficacy end points included major adverse cardiac events (MACE; cardiac death, myocardial infarction, or revascularization), left ventricular imaging, and other clinical-event surrogates. Safety and MACE were evaluated for up to 3 years. MPC injections were feasible and safe. Adverse events were similar across groups. No clinically symptomatic immune responses were noted. MACE was seen in 15 patients: 10 of 45 (22%) MPC-treated and 5 of 15 (33%) control patients. We found no differences between MPC-treated and control patients in survival probability, MACE-free probability, and all-cause mortality. We conducted a post hoc analysis of HF-related MACE (HF hospitalization, successfully resuscitated cardiac death, or cardiac death) and events were significantly reduced in the 150 million MPC group (0/15) versus control (5/15; 33%), 25 million MPC group (3/15; 20%), and 75 million MPC group (6/15; 40%); the 150 million MPC group differed significantly from all groups according to Kaplan-Meier statistics >3 years (P=0.025 for 150 million MPC group versus control).
Conclusions:
Transendocardial injections of allogeneic MPCs were feasible and safe in chronic HF patients. High-dose allogeneic MPCs may provide benefits in this population.
