Stem Cell–Derived Organoids as a Next‐Generation Platform for Drug Toxicity and Efficacy Testing: Toward Replacing Animal Models in Regulatory Science
Abstract
Three‐dimensional, self‐organizing structures derived from stem cells, known as organoids, represent a groundbreaking advancement in preclinical drug development. Organoid‐based platforms advance preclinical testing by providing an accurate representation of human tissue architecture and genetics, surpassing traditional two‐dimensional cultures and animal models in testing both drug safety and efficacy. Researchers are shifting toward organoid‐based systems as primary components of new approach methodologies, as global regulatory bodies increasingly acknowledge animal testing limitations. This review delivers an exhaustive examination of organoid technologies and their applications in drug testing. Our study explores current methods used to model toxic responses in different organs—such as the liver, kidney, and heart—while highlighting how personalized and disease‐specific organoids can enhance the accuracy of efficacy testing. Our investigation also examines regulatory frameworks and outlines the path toward organoid platform standardization and validation before their integration into drug development processes. Complex neural organoids show great promise but continue to face significant challenges, including biological variability, a lack of universal standards, and ethical concerns. The combination of organoid technology with microengineering techniques, artificial intelligence–based analysis, and high‐throughput screening methods represents a transformative change in…
How strong is the evidence?
Evidence Hub reads this as a review, done in animal and cell cultures (in vitro), not in people. Its evidence level is 5 of 5 (Very low · case / narrative / animal / in vitro / in silico). Case reports, narrative reviews, and animal, cell or computer-model studies give the weakest evidence for human health.
Read the paper
Related studies
- Safety and efficacy of COVID‐19 vaccines: A systematic review and meta‐analysis of controlled and randomized clinical trials
- Accuracy Comparison between Robot-Assisted Dental Implant Placement and Static/Dynamic Computer-Assisted Implant Surgery: A Systematic Review and Meta-Analysis of In Vitro Studies
- Determinants of passive antibody efficacy in SARS-CoV-2 infection: a systematic review and meta-analysis
- Cleaning efficacy of EDDY versus ultrasonically-activated irrigation in root canals: a systematic review and meta-analysis
- Bonding Efficacy of Universal Resin Adhesives to Zirconia Substrates: Systematic Review and Meta-Analysis
- In vitro antimicrobial activity of extracts and essential oils of Cinnamomum, Salvia, and Mentha spp. against foodborne pathogens: A meta-analysis study
Go deeper in Evidence Hub
Evidence Hub adds a study data sheet (design, sample size, evidence level, key numbers), an AI summary and a chat grounded in the paper's text.