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Current Medicinal Chemistry


ISSN (Print): 0929-8673
ISSN (Online): 1875-533X

Review Article

Induced Pluripotent Stem Cells as a Model for Therapy Personalization of Pediatric Patients: Disease Modeling and Drug Adverse Effects Prevention

Author(s): Elena Genova, Marco Pelin, Katsunori Sasaki*, Fengming Yue, Gaetana Lanzi, Stefania Masneri, Alessandro Ventura, Gabriele Stocco and Giuliana Decorti

Volume 25, Issue 24, 2018

Page: [2826 - 2839] Pages: 14

DOI: 10.2174/0929867324666170804150131

Price: $65


Understanding the biological and molecular processes underlying human pathologies is fundamental in order to develop innovative approaches to treat or prevent them. Among the technologies that could provide innovative disease models, induced pluripotent stem cells (iPSCs) is one of the most promising. Indeed, one application of this technology is patient-specific disease modeling. iPSCs obtained by reprogramming patients’ cells collected from accessible tissues, have the unique capability to differentiate, under an adequate stimulus, into any human cell type. In particular, iPSCs technology can be applied to study drug adverse effects, that is a key part of the drug discovery process. Indeed, drug induced adverse effects are among the most common causes that lead to abandon the development of new candidate therapeutic molecules, increasing the cost of drug discovery. An innovative strategy that could be used in drug design to solve drug attrition rate, and to establish innovative pharmacological models, could be the application of iPSCs technology in the early stage of the drug discovery process to model druginduced adverse events. In this review, recently developed disease models based on iPSCs will be discussed, with a particular focus on available models of drugs’ adverse effect, in particular hepatic/pancreatic toxicity.

Keywords: Induced pluripotent stem cells, disease modeling, drug adverse effects, pediatric patients, therapy personalization, innovative pharmacological models, hepatotoxicity, pancreatitis.

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