Human Induced Pluripotent Stem Cells: Role in Patient-Specific Drug Discovery

  • Michael Xavier Doss
  • Charles Antzelevitch
  • Agapios Sachinidis
Part of the Stem Cells and Cancer Stem Cells book series (STEM, volume 3)


Human in vitro models of inherited diseases that had never been possible earlier due to ethical and technical problems have been established recently by generating induced pluripotent stem (iPS) cells via reprogramming of patients’ own somatic cells such as dermal fibroblasts, adipocytes, and blood lymphocytes. The list of the established patient- and disease- specific iPS cells is expanding rapidly and is leading to an explosion of knowledge in understanding the novel pathophysiology of each of these diseases and in accelerating discovery of patient-specific drugs. It has been touted that the preliminary high throughput screening of novel drugs first on iPS cell based human in vitro models of diseases will cut down enormously the cost of drug development in the pharmaceutical industry by eliminating the frequent failed translation of drug trials from animal models to humans due to species difference and their unique robustness. It is possible that the variability of drug efficacies and drug side effects/toxicities due to genetic/ethnic differences may be better resolved with the use of iPS cells based in vitro models, thereby making the treatments to be tailored “specifically” to individual patients toward establishing of “personalized medicine”. This chapter summarizes the latest developments with iPS cell based in vitro disease models and their potential insurmountable impact on the pharmaceutical industry for discovery of cost-effective and less time-consuming drugs applicable for treatment of patient- specific diseases.


hiPS Somatic cells Drug Phenotypes Teratoma Toxicology 


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Copyright information

© Springer Science+Business Media B.V. 2012

Authors and Affiliations

  • Michael Xavier Doss
    • 1
  • Charles Antzelevitch
    • 2
  • Agapios Sachinidis
    • 3
  1. 1.Stem Cell CenterMasonic Medical Research LaboratoryUticaUSA
  2. 2.Masonic Medical Research LaboratoryUticaUSA
  3. 3.Center of Physiology and Pathophysiology, Institute of NeurophysiologyUniversity of CologneCologneGermany

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