Tumorigenicity assay essential for facilitating safety studies of hiPSC-derived cardiomyocytes for clinical application

Sci Rep. 2019 Feb 13;9(1):1881. doi: 10.1038/s41598-018-38325-5.

Abstract

Transplantation of cardiomyocytes (CMs) derived from human induced pluripotent stem cells (hiPSC-CMs) is a promising treatment for heart failure, but residual undifferentiated hiPSCs and malignant transformed cells may lead to tumor formation. Here we describe a highly sensitive tumorigenicity assay for the detection of these cells in hiPSC-CMs. The soft agar colony formation assay and cell growth analysis were unable to detect malignantly transformed cells in hiPSC-CMs. There were no karyotypic abnormalities during hiPSCs subculture and differentiation. The hiPSC markers TRA1-60 and LIN28 showed the highest sensitivity for detecting undifferentiated hiPSCs among primary cardiomyocytes. Transplantation of hiPSC-CMs with a LIN28-positive fraction > 0.33% resulted in tumor formation in nude rats, whereas no tumors were formed when the fraction was < 0.1%. These findings suggested that combination of these in vitro and in vivo tumorigenecity assays can verify the safety of hiPSC-CMs for cell transplantation therapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Carcinogenicity Tests / methods*
  • Cell Differentiation
  • Cell Proliferation
  • Cells, Cultured
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Induced Pluripotent Stem Cells / metabolism
  • Karyotype
  • Membrane Glycoproteins / metabolism
  • Myocytes, Cardiac / cytology*
  • Myocytes, Cardiac / metabolism
  • Myocytes, Cardiac / transplantation
  • Neoplasms / etiology
  • RNA-Binding Proteins / metabolism
  • Rats
  • Rats, Nude
  • Transplantation / adverse effects

Substances

  • Lin28A protein, human
  • Membrane Glycoproteins
  • RNA-Binding Proteins
  • endoplasmin