Long-term engraftment and maturation of autologous iPSC-derived cardiomyocytes in two rhesus macaques

Cell Stem Cell. 2024 Jul 5;31(7):974-988.e5. doi: 10.1016/j.stem.2024.05.005. Epub 2024 Jun 5.

Abstract

Cellular therapies with cardiomyocytes produced from induced pluripotent stem cells (iPSC-CMs) offer a potential route to cardiac regeneration as a treatment for chronic ischemic heart disease. Here, we report successful long-term engraftment and in vivo maturation of autologous iPSC-CMs in two rhesus macaques with small, subclinical chronic myocardial infarctions, all without immunosuppression. Longitudinal positron emission tomography imaging using the sodium/iodide symporter (NIS) reporter gene revealed stable grafts for over 6 and 12 months, with no teratoma formation. Histological analyses suggested capability of the transplanted iPSC-CMs to mature and integrate with endogenous myocardium, with no sign of immune cell infiltration or rejection. By contrast, allogeneic iPSC-CMs were rejected within 8 weeks of transplantation. This study provides the longest-term safety and maturation data to date in any large animal model, addresses concerns regarding neoantigen immunoreactivity of autologous iPSC therapies, and suggests that autologous iPSC-CMs would similarly engraft and mature in human hearts.

Keywords: NHP; NIS; PET/CT imaging; autologous transplantation; cardiac cellular therapy; chronic cardiac ischemic damage; iPSC; induced pluripotent stem cell; nonhuman primates; sodium/iodide symporter.

MeSH terms

  • Animals
  • Cell Differentiation
  • Humans
  • Induced Pluripotent Stem Cells* / cytology
  • Induced Pluripotent Stem Cells* / metabolism
  • Macaca mulatta*
  • Myocardial Infarction / pathology
  • Myocardial Infarction / therapy
  • Myocytes, Cardiac* / cytology
  • Myocytes, Cardiac* / metabolism
  • Positron-Emission Tomography
  • Time Factors
  • Transplantation, Autologous