干细胞治疗的前景 The Promise and Perils of Stem Cell Therapeutics.pdf

Cell Stem Cell PerspectiveThe Promise and Perils of Stem Cell TherapeuticsGeorge Q. Daley1,2,3,* 1Stem Cell Transplantation Program, Division of Pediatric Hematology/Oncology, Manton Center for Orphan Disease Research, Howard Hughes Medical Institute, Children’s Hospital Boston and Dana Farber Cancer Institute; Division of Hematology, Brigham and Women’s Hospital; and Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Boston, MA 02115, USA 2Broad Institute, Cambridge, MA 02142, USA 3Harvard Stem Cell Institute; Boston, MA 02138, USA *Correspondence: george.daley@ DOI 10.1016/j.stem.2012.05.010 Stem cells are the seeds of tissue repair and regeneration and a promising source for novel therapies. However, apart from hematopoietic stem cell (HSC) transplantation, essentially all other stem cell treatments remain experimental. High hopes have inspired numerous clinical trials, but it has been difficult to obtain unequivocal evidence for robust clinical benefit. In recent years, unproven therapies have been widely prac- ticed outside the standard clinical trial network, threatening the cause of legitimate clinical investigation. Numerous challenges and technical barriers must be overcome before novel stem cell therapies can achieve meaningful clinical impact.Cell Therapeutics: The Current Standard of Care In the twentieth century small molecule and protein drugs proved remarkably successful in restoringhealth andextending life span, but in the twenty-first century our aging population will face an increasing burden of organ failure and neurodegenerative disease. Such conditions are unlikely to be cured by drugs alone and instead call for restoration of tissue function through novel therapeutic approaches. Transplantation of whole organs— heart, lung, liver, kidney, small bowel, and pancreas—has become routine in modern medicine and has saved countless lives, while grafts of the skin and cornea for burns or ocular injury and transfusion