Recently, Dr. YANG Huangtian's team from the Shanghai Institute of Nutrition and Health (SINH) of the Chinese Academy of Sciences (CAS) and Dr. CAO Nan's team from Sun Yat-sen University reported a perspective article on human pluripotent stem cell-derived cardiomyocytes in heart disease modeling and repair. The study was published in Stem Cells Translational Medicine on July 29.

Heart diseases (HDs) encompass a broad range of disorders extending from myocardial infarction (MI) to heritable cardiomyopathies, and heart failure (HF) is the leading cause of morbidity and mortality.

Cell therapy by transplantation of human pluripotent stem cells (hPSCs)-derived cardiac lineage cells is one of the most notable alternative therapeutic approaches. hPSCs, including embryonic stem cells (hESCs) and induced pluripotent stem cells (hiPSCs), have the potential to differentiate into cardiac lineage cells.

Preclinical studies by Dr. YANG Huangtian's research team from SINH revealed that hPSC-derived cardiac lineage cells have the potential to differentiate into functional cells to replace lost cells after myocardial infarction.

Meanwhile, transplantation of hPSC-derived cardiac lineage cells contributed to infarct healing via trigger endogenous cardiac repair mechanisms. In addition, no tumorigenesis and arrhythmia had been observed in the preliminary clinical trials for patients with severe ischemic heart failure.  

HPSC-based disease modeling has not only shed new lights on the genetic and/or molecular basis of many cardiac diseases, but also established cellular phenotypes characterizing HDs, revealing tremendous potential in the phenotypic approach of drug discovery.

Although the outcomes are encouraging, several obstacles, such as cell type, maturity, quality control, residence, immune rejection and arrhythmia induced by cell transplantation need to be overcome before moving toward clinical application. 

This work was supported by the National Natural Science Foundation of China, the National Key R&D Program of China, Strategic Priority Research Program of CAS, the Guangdong Innovative and Entrepreneurial Research Team Program, and the Fundamental Research Funds for the Central Universities.