Lalit Pratik A, Hei Derek J, Raval Amish N, Kamp Timothy J
From the Department of Medicine (P.A.L., A.N.R., T.J.K.), Molecular and Cellular Pharmacology Program (P.A.L., T.J.K.), and Stem Cell and Regenerative Medicine Center (P.A.L., D.J.H., A.N.R., T.J.K.), Waisman Biomanufacturing at University of Wisconsin, Madison (D.J.H.).
Circ Res. 2014 Apr 11;114(8):1328-45. doi: 10.1161/CIRCRESAHA.114.300556.
Coronary artery disease with associated myocardial infarction continues to be a major cause of death and morbidity around the world, despite significant advances in therapy. Patients who have large myocardial infarctions are at highest risk for progressive heart failure and death, and cell-based therapies offer new hope for these patients. A recently discovered cell source for cardiac repair has emerged as a result of a breakthrough reprogramming somatic cells to induced pluripotent stem cells (iPSCs). The iPSCs can proliferate indefinitely in culture and can differentiate into cardiac lineages, including cardiomyocytes, smooth muscle cells, endothelial cells, and cardiac progenitors. Thus, large quantities of desired cell products can be generated without being limited by cellular senescence. The iPSCs can be obtained from patients to allow autologous therapy or, alternatively, banks of human leukocyte antigen diverse iPSCs are possible for allogeneic therapy. Preclinical animal studies using a variety of cell preparations generated from iPSCs have shown evidence of cardiac repair. Methodology for the production of clinical grade products from human iPSCs is in place. Ongoing studies for the safety of various iPSC preparations with regard to the risk of tumor formation, immune rejection, induction of arrhythmias, and formation of stable cardiac grafts are needed as the field advances toward the first-in-man trials of iPSCs after myocardial infarction.
尽管治疗方面取得了重大进展,但冠心病合并心肌梗死仍是全球死亡和发病的主要原因。发生大面积心肌梗死的患者发生进行性心力衰竭和死亡的风险最高,而基于细胞的疗法为这些患者带来了新希望。由于将体细胞重编程为诱导多能干细胞(iPSC)取得了突破,一种最近发现的用于心脏修复的细胞来源应运而生。iPSC可以在培养物中无限增殖,并能分化为心脏谱系细胞,包括心肌细胞、平滑肌细胞、内皮细胞和心脏祖细胞。因此,可以大量生成所需的细胞产物,而不受细胞衰老的限制。iPSC可以从患者自身获取以进行自体治疗,或者,也可以建立人类白细胞抗原多样的iPSC库用于异体治疗。使用从iPSC生成的各种细胞制剂进行的临床前动物研究已显示出心脏修复的证据。从人类iPSC生产临床级产品的方法已经具备。随着该领域向心肌梗死后iPSC的首次人体试验迈进,需要持续开展各项研究,以评估各种iPSC制剂在肿瘤形成风险、免疫排斥、心律失常诱导以及稳定心脏移植物形成方面的安全性。