Accelerating drug discovery with organoids
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Part of series
Pushing the boundaries of science starts here
Scientists at Roche and Hubrecht Institute review progress in the field and exciting future opportunities to apply human models in industry settings.
The future of faster drug discovery may already be on the horizon. Thanks to a relatively new model of human biology known as organoids — structures that resemble, but are simpler than, human organs, and can be grown in a lab from human cells — it’s now possible to develop, test, evaluate efficacy and safety, and deliver a molecule to human patients in a clinical trial much more quickly than before. “For example, one antibody went from an idea to human patients in two and a half years, based on organoid testing alone, with no animal or cell lines involved,” said Hans Clevers, ad interim Director of Roche’s Institute of Human Biology (IHB) and distinguished group leader at the Hubrecht Institute. “The result was a safe, effective molecule now in phase 3 clinical trials.” Today, this example is still an exception, but scientists are hard at work trying to make it the norm.
:quality(90)/ "3D-cultured human intestinal organoid with BEST4⁺ cells and goblet cell mucins labeled in magenta and green, respectively. Credit: Daisong Wang")
Organoids have historically been used in basic research settings to uncover new biology. In a recent review appearing in Nature Reviews Drug Discovery, Clevers and colleagues from Roche and the Hubrecht Institute highlight how organoids have been or could be used all along the drug discovery pipeline. “This review shows that organoids can already do many things that can improve drug development,” said first author Daisong Wang, Postdoctoral Researcher at the Hubrecht Institute.
In writing the review, the authors were surprised to find that there are not yet many published use cases for organoids outside of early biological discovery. But by highlighting the applications to date, the authors hope to address some of the concerns of applying organoids in the drug discovery pipeline. “By showcasing successful applications, this review could increase confidence in organoid-based approaches and accelerate their adoption across the pharmaceutical industry,” said co-author Rémi Villenave, Principal Scientist in Roche’s department Pharmaceutical Sciences. One particularly promising area for organoids that the authors highlight is pharmacology, or understanding the properties of medicines in living organisms.
:quality(90)/ "2D-cultured human hepatocytes derived from organoids. Normal hepatocytes are labeled in red, and hepatocytes harboring oncogenic mutations are labeled in green within the co-culture. Credit: Daisong Wang")
The review comes at a time when the field is gaining momentum. Research and application is moving beyond academic groups thanks to cross-disciplinary research initiatives like IHB, who are helping to innovate organoid technology for drug development use. At the same time, regulatory agencies across the world are showing greater interest in leveraging organoid-based data for regulatory submissions. The comprehensive review provides a starting place for drug developers to see how to use organoids to study diseases and develop medicines to help patients.
“Organoid technology is a revolution that we’ve been dreaming of for a long time,” said co-author Nadine Stokar-Regenscheit, Pathologist in Roche’s Pharmaceutical Sciences. “We have an opportunity to show people that it’s ready, and guide the discussion on how to use it.”
“There’s nothing to lose by exploring organoids in drug discovery — we can only gain,” she concluded.
Reflections from the authors
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Organoid models enable direct exploration of human biology, and may revolutionize our understanding and treatment of disease.
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There’s nothing to lose by exploring organoids in drug discovery — we can only gain. As interdisciplinary science teams developing drugs for patients, it is now our responsibility to explore how organoids/NAMs can most effectively support our preclinical drug development and increase its predictivity towards patients.
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We are witnessing a pivotal transition: fit-for-purpose organoid/NAMs applications across the industry are beginning to complement - and in some contexts reduce reliance on conventional approaches, supported by regulatory and political momentum. These are exciting times to work in this space and contribute to its advancement.
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Our review shows how organoid and stem-cell models are helping make drug discovery smarter and faster. But there’s still a long way to go — from making the models more consistent to proving they really predict what happens in patients.
Journal papers are available open access:
Wang D*, Stokar-Regenscheit N, Villenave R, Clevers H^ (2025). Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges. Nature Reviews Drug Discovery. https://rdcu.be/ePxiy
*lead authors
^senior corresponding authors
About the image (at top):
Color-tagging shows how an organoid can recreate distinct cell composition found in the native organ. Specifically, a human duodenum organoid displaying six different genetically encoded fluorescent labels or tags. Enterocyte cells are marked in red, enteroendocrine cells (EECs) in magenta, goblet cells in blue, Paneth cells in green, E-cadherin in yellow and all nuclei in turquoise. The organoid’s coloration demonstrates patterning of cells as they would be found in vivo, with enterocytes only found in the lumen, and goblet cells and EECs scattered throughout. Credit, Ninouk Akkerman, Yannik Bollen, Jannika Finger and Rya Riedweg (IHB)