Stem Cells and Organoids as Tools for Drug Development

Dear Colleagues,

In the last 20 years, pharmaceutical development and evaluation costs have increased dramatically. Numerous failed clinical trials indicate that drug development and testing procedures in animal models and standard cell lines are not reliably predictive for human efficacy and safety.

Human embryonic stem cells (hESCs) and human-induced pluripotent stem cells (hiPSCs) can be propagated extensively in vitro while retaining the ability to differentiate into any specialized cell type within the body; these unique features of hPSCs offer a way of producing relevant in vitro models amenable to high-throughput testing for drug discovery. Moreover, adult PSCs and iPSCs derived from patient tissues may not only be a source of cells for autologous therapies, but also for individual customized in vitro drug testing and studies on the molecular mechanisms of disease.

In the last decade, organoid technology, an innovative three-dimensional (3D) model, has risen rapidly and become increasingly prevalent among researchers. Organoids are 3D tissues in miniature in vitro (established from ESCs, iPSCs, and tumor cells), which are specific to the parent counterparts in vivo. Organoid technology demonstrates tremendous potential for clinical applications. Firstly, organoids can be exploited as an excellent platform to evaluate drug efficacy and toxicity, ultimately promoting drug development. Secondly, precision medicine may represent another pivotal branch of organoid technology by accurately predicting drug responses and guiding optimized treatment strategies for each individual patient.

This Special Issue will provide an opportunity to discuss the application of stem cells and organoids from healthy and diseased sources in drug screening and their role in complementing, reducing, and replacing animal trials. In addition to this, target identification and major advances in the field of personalized medicine using induced pluripotent cells will also be focused on. We welcome articles that deal with all aspects of pharmaceutical and medical care, from basic to clinical research, and invite scientists to publish their original research works, review articles, and communications on this wide health domain.

Dr. Daniela Omodei
Guest Editor

Manuscript Submission Information

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• stem cells
• patient-derived cells
• iPSCs (induced pluripotent stem cells)
• organoids
• drug development
• drug screening
• drug toxicity
• preclinical and clinical drug research
• novel therapeutics
• disease modeling
• personalized medicine
• precision medicine