Current and Future Applications of Three-Dimensional Brain and Cardiac Organoids in Translational Medicine: From Disease Modeling to Drug Discovery

Over the past decade, organoid research has made transformative advances, emerging as a powerful platform to address key limitations of traditional biomedical models. Although animal systems remain indispensable for studying disease mechanisms, their limited ability to accurately recapitulate human-specific physiology and pathophysiology has contributed to the high failure rate of drug candidates during clinical translation. The emergence of three-dimensional (3D) organoid systems, such as brain and cardiac organoids derived from stem cells, represents a major technological breakthrough. These self-organizing multicellular constructs closely mimic key architectural, cellular, and functional features of native human tissues, enabling more physiologically relevant modeling of complex neurological and cardiovascular disorders. Beyond fundamental biological investigations, brain and cardiac organoids have demonstrated substantial utility in drug screening, toxicity assessment, and precision medicine approaches, including patient-specific disease modeling and therapeutic response prediction. This review highlights recent progress in brain and cardiac organoid technologies, discusses their applications in translational and regenerative medicine, and evaluates their current limitations and future directions in disease modeling and drug discovery.

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Publication Details

Journal
ACS Pharmacology & Translational Science
Published
2026-02-16
DOI
https://doi.org/10.1021/acsptsci.5c00630
Primary Topic
Pluripotent Stem Cells Research
Type
article
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article

Current and Future Applications of Three-Dimensional Brain and Cardiac Organoids in Translational Medicine: From Disease Modeling to Drug Discovery

Dhiraj Devidas Bhatia, Raghu Solanki, Anchal Kaushik, Daby Thakur Prasad Mishra
ACS Pharmacology & Translational Science
Pluripotent Stem Cells Research
article

Current and Future Applications of Three-Dimensional Brain and Cardiac Organoids in Translational Medicine: From Disease Modeling to Drug Discovery

Dhiraj Devidas Bhatia, Raghu Solanki, Anchal Kaushik, Daby Thakur Prasad Mishra
article en

Abstract

Over the past decade, organoid research has made transformative advances, emerging as a powerful platform to address key limitations of traditional biomedical models. Although animal systems remain indispensable for studying disease mechanisms, their limited ability to accurately recapitulate human-specific physiology and pathophysiology has contributed to the high failure rate of drug candidates during clinical translation. The emergence of three-dimensional (3D) organoid systems, such as brain and cardiac organoids derived from stem cells, represents a major technological breakthrough. These self-organizing multicellular constructs closely mimic key architectural, cellular, and functional features of native human tissues, enabling more physiologically relevant modeling of complex neurological and cardiovascular disorders. Beyond fundamental biological investigations, brain and cardiac organoids have demonstrated substantial utility in drug screening, toxicity assessment, and precision medicine approaches, including patient-specific disease modeling and therapeutic response prediction. This review highlights recent progress in brain and cardiac organoid technologies, discusses their applications in translational and regenerative medicine, and evaluates their current limitations and future directions in disease modeling and drug discovery.

ACS Pharmacology & Translational ScienceVol. 9(3)
Indian Institute of Technology Gandhinagar (IN)
Good health and well-being
Openalex Percentile: Top 54%
Pluripotent Stem Cells Research
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