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| The Prospect of Induction of Pluripotent Stem Cell Technology in Drug Research and Development |
| WU Sheng-xing1, LI Yan3, ZHANG Hai-yan1, LIU Yang1, LAI Qiong1, YANG Ming1,2 |
1. Key Laboratory of Modern Preparation of TCM, Ministry of Education, Jiangxi University of TCM, Nanchang 330004, China;
2. Chengdu University of TCM, Chengdu 610075, China;
3. National Engineering Research Center for Traditional Chinese Medicine Solid Chemicals Manufacturing, Jiangxi University of Traditional Chinese Medicine, Nanchang 330004, China |
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Abstract Despite continuous efforts to improve the process of drug discovery and development,achieving success at the clinical stageremains challenging because of a persistent translational gap between the preclinical and clinical settings. Under these circumstances, the discovery of human induced pluripotent stem (iPS) cells has brought new hope to the drug discovery field because they enable scientists to humanize a variety of pharmacological and toxicological models in vitro.Theavailability of human iPS cell-derived cells,their potential to differentiate into specific functional cells, tissues and organs, has provided new avenues for studying diseases and cell therapy. Biomarkers are an essential part of the translational effort to shift new discoveries from bench to bedside as they provide a measurable indicator to evaluate pharmacological and toxicological effects in both preclinical and clinical settings. In general, during the preclinical stage of drug development, in vitro models that are established to recapitulate human diseases are validated by using a set of biomarkers; however, their translatability to a clinical setting remains problematic. An overview of the current strategies for human iPS cell-based drug discovery from the perspective of translational research is provided.
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Received: 17 May 2017
Published: 15 November 2017
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| [1] |
Caulfield T, Sipp D, Murry C E, et al. SCIENTIFIC COMMUNITY. Confronting stem cell hype. Science, 2016, 352(6287):776.
|
|
|
| [2] |
Avior Y, Sagi I, Benvenisty N. Pluripotent stem cells in disease modelling and drug discovery. Nature Reviews Molecular Cell Biology, 2016, 17(3):170.
|
|
|
| [3] |
Wu Y, Li O, He C, et al. Generation and characterization of induced pluripotent stem cells from guinea pig fetal fibroblasts. Molecular Medicine Reports, 2017, 15(6):3690-3698.
|
|
|
| [4] |
Spitalieri P, Talarico V, Luchetti A, et al. Generation of disease-specific induced pluripotent stem cells from human fetal extra-embryonic tissues. Cytotherapy, 2014, 16(4):S9-S9.
|
|
|
| [5] |
Singh A M, Perry D W, Steffey V V A, et al. Decoding the epigenetic heterogeneity of human pluripotent stem cells with seamless gene editing. Methods in Molecular Biology, 2016, 1516:153.
|
|
|
| [6] |
Faulknerjones A, Fyfe C, Cornelissen D J, et al. Bioprinting of human pluripotent stem cells and their directed differentiation into hepatocyte-like cells for the generation of mini-livers in 3D. Biofabrication, 2015, 7(4):044102.
|
|
|
| [7] |
Smietana K, Siatkowski M, Møller M. Trends in clinical success rates. Nature Reviews Drug Discovery, 2016, 15(6):379.
|
|
|
| [8] |
De V J, Bouckenheimer J, Sansac C, et al. Human induced pluripotent stem cells:A disruptive innovation. Current Research in Translational Medicine, 2016, 64(2):91-96.
|
|
|
| [9] |
Mullane K, Winquist R J, Williams M. Translational paradigms in pharmacology and drug discovery. Biochemical Pharmacology, 2014, 87(1):189-210.
|
|
|
| [10] |
Zhao X, Modur V, Carayannopoulos L N, et al. Biomarkers in pharmaceutical research. Clinical Chemistry, 2015, 61(11):1343-1353.
|
|
|
| [11] |
Quintana-Bustamante O, Segovia J C. Generation of patient-specific induced pluripotent stem cell from peripheral blood mononuclear cells by sendai reprogramming vectors. Methods in Molecular Biology, 2016, 1353:1-11.
|
|
|
| [12] |
Spitalieri P, Talarico V, Luchetti A, et al. Generation of disease-specific induced pluripotent stem cells from human fetal extra-embryonic tissues. Cytotherapy, 2014, 16(4):S9-S9.
|
|
|
| [13] |
Narayanan G, Sheila M, Chai J, et al. Generation of sibling-matched induced pluripotent stem cell lines from spinal and bulbar muscular atrophy patients. Stem Cell Research, 2017, 20:30-33.
|
|
|
| [14] |
Slaugenhaupt S A, Gusella J F. Methods for Altering mRNA Splicing and Treating Familial Dysautonomia by Administering Benzyladenine:US,9265766 B2. 2016-02-23.
|
|
|
| [15] |
Inoue H, Nagata N, Kurokawa H, et al. iPS cells:a game changer for future medicine. Embo Journal, 2014, 33(5):409.
|
|
|
| [16] |
Zhang Z N, Freitas B C, Qian H, et al. Layered hydrogels accelerate iPSC-derived neuronal maturation and reveal migration defects caused by MeCP2 dysfunction. Proceedings of the National Academy of Sciences of the United States of America, 2016, 113(12):3185.
|
|
|
| [17] |
Tang X, Kim J, Zhou L, et al. KCC2 rescues functional deficits in human neurons derived from patients with Rett syndrome.. Proceedings of the National Academy of Sciences of the United States of America, 2016, 113(3):751.
|
|
|
| [18] |
Tanaka T, Takahashi K, Yamane M, et al. Induced pluripotent stem cells from CINCA syndrome patients as a model for dissecting somatic mosaicism and drug discovery. Blood, 2012, 120(6):1299.
|
|
|
| [19] |
Liu G H, Suzuki K, Qu J, et al. Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs. Cell Stem Cell, 2011, 8(6):688-694.
|
|
|
| [20] |
Bannon D, Landau A M, Doudet D J. How relevant are imaging findings in animal models of movement disorders to human disease? Current Neurology & Neuroscience Reports, 2015, 15(8):53.
|
|
|
| [21] |
Suarez C D, Littlepage L E. Patient-derived tumor xenograft models of breast cancer. Methods MolBiol, 2016, 1406:211-223.
|
|
|
| [22] |
Rinaldi F, Perlingeiro R C. Stem cells for skeletal muscle regeneration:therapeutic potential and roadblocks. Translational Research, 2014, 163(4):409-417.
|
|
|
| [23] |
Cevatemre B, Ulukaya E, Sarimahmut M, et al. The M30 assay does not detect apoptosis in epithelial-derived cancer cells expressing low levels of cytokeratin 18. Tumor Biology, 2015, 36(9):6857-6865.
|
|
|
| [24] |
Öngen Z. What do biomarkers mark? Anatolian Journal of Cardiology, 2016, 16(2):75.
|
|
|
| [25] |
Ding Y, Qin Z H, Cun-Shuan X U. Generation of hepatocyte-like cells from induced pluripotent stem cells. Chinese Journal of Biochemistry & Molecular Biology, 2016,32(1):41-48.
|
|
|
| [26] |
Swinney D C, Anthony J. How Were New Medicines Discovered? Nature Reviews Drug Discovery, 2011, 10(7):507-519.
|
|
|
| [27] |
Lee H, Lee J W. Target identification for biologically active small molecules using chemical biology approaches. Archives of Pharmacal Research, 2016, 39(9):1-9.
|
|
|
| [28] |
Wagner B, Schreiber S. The power of sophisticated phenotypic screening and modern mechanism-of-action methods. Cell Chemical Biology, 2016, 23(1):3-9.
|
|
|
| [29] |
Kobari L, Yates F, Oudrhiri N, et al. Human induced pluripotent stem cells can reach complete terminal maturation:in vivo and in vitro evidence in the erythropoietic differentiation model. Haematologica, 2012, 97(12):1795-803.
|
|
|
| [30] |
Janzen W P. Screening technologies for small molecule discovery:The State of the Art. Chemistry & Biology, 2014, 21(9):1162-1170.
|
|
|
| [31] |
Tang S, Xie M, Cao N, et al. Patient-specific induced pluripotent stem cells for disease modeling and phenotypic drug discovery. Journal of Medicinal Chemistry, 2016, 59(1):2.
|
|
|
| [32] |
Lee W M. Drug-induced hepatotoxicity. New England Journal of Medicine, 2010, 7(5):477-485.
|
|
|
| [33] |
Chen M, Suzuki A, Borlak J, et al. Drug-induced liver injury:interactions between drug properties and host factors. Journal of Hepatology, 2015, 63(2):503-514.
|
|
|
| [34] |
Tasnim F, Toh Y C, Qu Y, et al. Functionally enhanced human stem cell derived hepatocytes in galactosylated cellulosic sponges for hepatotoxicity testing. Molecular Pharmaceutics, 2016, 13(6):1947.
|
|
|
| [35] |
Zhu S, Rezvani M, Harbell J, et al. Mouse liver repopulation with hepatocytes generated from human fibroblasts. Nature, 2014, 508(7494):93.
|
|
|
| [36] |
Hay M, Thomas D W, Craighead J L, et al. Clinical development success rates for investigational drugs. Nature Biotechnology, 2014, 32(1):40.
|
|
|
| [37] |
Vernetti L A, Senutovitch N, Boltz R, et al. A human liver microphysiology platform for investigating physiology, drug safety, and disease models. Experimental Biology & Medicine, 2016, 241(1):101.
|
|
|
| [38] |
Schwartz M P, Hou Z, Propson N E, et al. Human pluripotent stem cell-derived neural constructs for predicting neural toxicity. Proceedings of the National Academy of Sciences of the United States of America, 2015, 112(40):12516.
|
|
|
| [39] |
Yu Y, Sun S, Wang S, et al. Liensinine-and neferine-induced cardiotoxicity in primary neonatal rat cardiomyocytes and human-induced pluripotent stem cell-derived cardiomyocytes. International Journal of Molecular Sciences, 2016, 17(2):186.
|
|
|
| [40] |
Wei D, Mak R H. Early markers of obesity-related renal injury in childhood. Pediatric Nephrology, 2015, 30(1):1-4.
|
|
|
| [41] |
Tiong H Y, Huang P, Xiong S, et al. Drug-induced nephrotoxicity:clinical impact and pre-clinical in vitro models. Molecular Pharmaceutics, 2014, 11(7):1933-1948.
|
|
|
| [42] |
Van M L, Moerland M, Cohen A F, et al. Urinary kidney biomarkers for early detection of nephrotoxicity in clinical drug development. British Journal of Clinical Pharmacology, 2014, 77(6):947-957.
|
|
|
| [43] |
Snoeck H W. Modeling human lung development and disease using pluripotent stem cells. Development, 2015, 142(1):13-16.
|
|
|
| [44] |
Tukker A M, de Groot M W, Wijnolts F M, et al. Is the time right for in vitro neurotoxicity testing using human iPSC-derived neurons? Altex, 2016, 33(3):261.
|
|
|
| [45] |
Hartmann N B, Ågerstrand M, Lützhøft H C H, et al. NanoCRED:A transparent framework to assess the regulatory adequacy of ecotoxicity data for nanomaterials-relevance and reliability revisited. Nanoimpact, 2017,6:81-89.
|
|
|
| [46] |
Aday S, Cecchelli R, Halliervanuxeem D, et al. Stem cell-based human blood-brain barrier models for drug discovery and delivery. Trends in Biotechnology, 2016, 34(5):382-393.
|
|
|
| [47] |
Wheeler H E, Wing C, Delaney S M, et al. Modeling chemotherapeutic neurotoxicity with human induced pluripotent stem cell-derived neuronal cells. PLoS One, 2015, 10(2):e0118020.
|
|
|
| [48] |
Sayed N, Liu C, Wu J C. Translation of human-induced pluripotent stem cells:from clinical trial in a dish to precision medicine. Journal of the American College of Cardiology, 2016, 67(18):2161-2176.
|
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