Applications of Stem Cells and Organoids in Disease Modeling, Drug Discovery and Clinical Evaluation

Dear Colleagues,

Stem cells and organoids are powerful tools that have significantly advanced disease modeling, drug discovery and the assessment of drug efficacy. Induced pluripotent stem cells (iPSCs), which can be reprogrammed from adult cells and differentiated into various tissue types, allow researchers to create disease-relevant human cell models for high-throughput drug screening. These models enable the identification of drug candidates that are more likely to succeed in clinical trials, thereby reducing costs and time in early-stage development.

Organoids, 3D structures derived from stem cells that mimic the architecture and function of human organs, provide a more physiologically relevant system for evaluating drug effects. Indeed, organoids derived from the liver, kidney, brain, and intestine can replicate key aspects of organ-specific metabolism, toxicity, and transport mechanisms. This makes them ideal for predicting a compound’s pharmacokinetics and safety profile before entering animal or human studies.

Moreover, in the biological evaluation phase, stem cell-derived cells and organoids offer insights into how drugs interact with human-specific targets, minimizing the translational gap often seen with animal models. They are particularly valuable in assessing off-target effects, dose-dependent toxicity, and mechanisms of action in a tissue-specific context. In addition, disease-specific organoids derived from patient iPSCs allow personalized assessment of therapeutic response. For example, cancer organoids (tumoroids) derived from patient tumors are now used to screen chemotherapy and targeted therapies, helping clinicians select the most effective treatment with minimal side effects. Similarly, neurological and cardiac organoids are being used to evaluate drug efficacy in treating diseases like epilepsy or cardiomyopathies.

This Special Issue will provide an opportunity to discuss the application of stem cells and organoids from healthy and diseased sources in drug development/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

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.