Lab-Grown Human Organoids Poised to Transform Drug Testing and Pandemic Preparedness
核心洞察
A transatlantic collaboration between the University of Calgary and the Doherty Institute (搜索) aims to standardize human lung and gut organoid protocols to improve clinical trial success rates.
UK scientists at Cambridge will build a library of standardized, validated organoids from NHS patient cells, funded by a £20 million Medical Research Council grant.
More than 90% of drugs that pass animal testing fail in human trials, underscoring the urgent need for more predictive human-relevant models.
A growing international push to replace animal models with lab-grown miniature human organs is gathering momentum, with two major initiatives on opposite sides of the globe aiming to standardize organoid technology for drug testing and pandemic preparedness.
More than 90% of drugs that clear animal testing go on to fail in human trials, a statistic that has long raised questions about the predictive value of traditional preclinical models. Now, researchers in the UK, Canada, and Australia are building standardized organoid platforms they believe will dramatically improve the translational pipeline.
The standardization challenge
Organoid technology has existed only since 2009, and while it has advanced rapidly, a fundamental obstacle has hindered global adoption: every laboratory uses its own protocols.
"Each lab is currently using its own recipe to grow organoids – and if we're all using different protocols, we can't collaborate, compare, or reproduce each other's results," said Dr Simon Hirota at the University of Calgary.
Dr Laura Cook at the Doherty Institute (搜索) in Melbourne added: "Science is grappling with a reproducibility crisis where a significant proportion of findings published in leading journals can't be independently replicated. In pandemic preparedness, where speed and confidence are critical, this is a serious problem."
Their transatlantic collaboration aims to harmonize gut and lung organoid protocols between the two laboratories, creating a shared, validated foundation for the global research community. The process involves rigorously testing every step, swapping tissue samples between Melbourne and Calgary, and confirming that both labs produce identical results from the same starting materials.
Integrating immune cells for host-directed therapeutics
The project adds complexity by incorporating immune cells into the organoid models – a critical feature for testing host-directed therapeutics, which modify the body's own immune response rather than targeting pathogens directly.
"You need the added immune cells to be able to test therapeutic activity on inflammatory responses," Dr Cook explained. "But immune cells can behave unpredictably in organoid conditions, requiring careful optimisation."
The team has spent four years refining these co-culture conditions and is now collaborating to establish standardized protocols. The goal is a validated gut and lung organoid platform that can be rapidly deployed in the event of another pandemic, enabling researchers worldwide to investigate disease pathways and screen potential therapeutics with confidence in cross-lab reproducibility.
A UK national organoid library
In parallel, the UK is making a substantial investment in organoid infrastructure. A new research hub in Cambridge, funded with £20 million from the Medical Research Council, will create a library of standardized, validated organoids available to academics and the pharmaceutical industry.
Scientists will grow the miniature tissues from NHS patients' cells, enabling them to study how diseases vary between individuals and identify which treatments work best for different patients based on their specific pathology.
"It's going to have a major impact on the numbers of animals used and the way we develop new drugs in the future," said Matthias Zilbauer, a clinical professor of paediatric gastroenterology at the Cambridge Stem Cell Institute (搜索). "We're not saying there won't be any animal use in the near or foreseeable future, because there are still certain issues that cannot be tested in these new models, but the reduction is very real."
Zilbauer's team is starting with organoids for inflammatory bowel diseases such as ulcerative colitis (搜索) and Crohn's disease (搜索), while other groups will focus on growing tumors to improve cancer treatments and brain organoids to understand neurological conditions.
The animal testing landscape
Last year, there were 2.54 million animal testing procedures in Britain, down 3.8% on 2024. More than 90% of those procedures used mice, rats, fish, and birds, while 1% used specially protected species such as cats, dogs, horses, and monkeys.
The UK government strategy, drawn up under Keir Starmer's administration, relies on "new approach methodologies" (NAMs) – including organoids, organ-on-a-chip systems, and artificial intelligence – to fast-track the reduction of animals in research.
A further £2 million has been awarded by Innovate UK (搜索) for nine projects aimed at reducing the use of animals such as dogs and monkeys in safety tests. One company, VivoSphere (搜索), is growing heart cells in tiny gel spheres for cardiac safety testing, where traditional approaches can involve 50 to 100 animals such as guinea pigs, rabbits, and dogs.
Beyond pandemic preparedness
The implications of standardized organoid platforms extend well beyond infectious disease outbreaks. Researchers envision applications in vaccine testing, personalized medicine, rare disease research, and a more reliable foundation for discovery and translational science globally.
"One of the huge advantages of organoids, organs-on-a-chip and other in vitro microphysiological systems is that, unlike animal models, they have real potential to deliver the promise of truly personalised medicine for individual patients," said Dr Juliet Dukes of the charity Replacing Animal Research. "It is all very exciting."
By establishing organoids as a standardized experimental technique, these initiatives aim to strengthen the translational pipeline, improving both the speed and likelihood with which new therapeutics progress from discovery through clinical trials and ultimately into patient care.