Cambridge will lead on a new £20million Pre-clinical Translational Models Hub that will develop next-generation human disease models, reducing the need for animal testing.

Funded by a £20million investment from the Medical Research Council (MRC), the hub will utilise stem cells, organoids and artificial intelligence to help transform how new medicines are created.

Human intestinal organoids. Image: University of Cambridge

The hub will build on the expertise of the Cambridge Stem Cell Institute (CSCI) and will be co-directed by Prof Matthias Zilbauer and Prof Bertie Göttgens.

Prof Zilbauer, group leader at the CSCI, said: "Over the past decade, we've seen the enormous potential of patient-derived organoids and other approaches that do not rely on the use of animals to transform the way we develop new medicines.

“Because these miniature human tissues retain many of the unique biological characteristics of the individual patient, they allow us to understand disease more accurately and test potential treatments before they ever reach the clinic. This has the potential to bring more effective, personalised treatments to patients while reducing the time and cost of developing new medicines and our reliance on animal models."

Organoids are three-dimensional tissue cultures grown in the lab from stem cells to act like mini organs for testing.

Prof Göttgens, director of the CSCI, said: "By combining expertise in stem cell biology, organoid technology and artificial intelligence, the new UK Pre-clinical Translational Models Hub will accelerate the development of next-generation human disease models. Together with our academic, NHS and industry partners, we aim to deliver better treatments for patients while reinforcing the UK's global leadership in this field."

AI-powered approaches, bioengineering and clinical research will be combined with CSCI’s capabilities in advanced human in vitro models, organoids and other stem cell-derived systems to create more accurate and predictive ways of studying human disease and testing potential treatments before they reach patients.

Partnerships with hospitals, research institutes and industry partners across Cambridge and the UK will be developed to establish a national network dedicated to developing, validating and accelerating the adoption of next-generation preclinical models.

The hub will allow researchers, clinicians and companies across the country to access these human-based platforms that better reflect human biology with the aim of improving the development of new therapies.

Treatments that appear promising in animal models often fail when it comes to testing in humans, often due to toxicity, meaning years of research and significant investment can be lost before a medicine ever reaches a patient.

But human cells and tissues - including organoids and other engineered human systems - plus computational models can be used to recreate aspects of disease in the laboratory, improving predictions of how patients may respond to new therapies.

Professor Bertie Göttgens, left, and Professor Matthias Zilbauer, from the Cambridge Stem Cell Institute (CSCI). Picture: CSCI

Such technologies have already transformed biomedical research but their use in drug development requires robust validation, standardisation and benchmarking to provide confidence that such models are reliable, reproducible and capable of improving decision-making.

The MRC says the Pre-clinical Translational Models Hub will help address this challenge by creating a national UK accelerator platform that will develop promising human-based models into validated, scalable and industry-ready technologies.

The field of stem cell biology emerged in 2009 and CSCI has played a leading role in developing organoids and other advanced human models since it was established in 2012.

Prof Zilbauer has built a biobank of more than 1,000 patient-derived organoid lines over the past decade.

Through clinical research into paediatric inflammatory bowel diseases at Cambridge University Hospitals, he works with children and their families to collect patient samples, generate organoid models, understand disease mechanisms and test potential new treatments.

“Patient-derived models have enormous potential to transform the way we understand disease and develop new medicines,” he said. “By capturing important characteristics of individual patients, these human models allow us to study disease more accurately and test potential treatments before they reach the clinic. This could ultimately lead to more effective, personalised therapies while reducing the time and cost of drug development.”

CSCI will work with affiliate group leaders and hub co-leads Dr Mathew Garnett and Dr Mo Lotfollahi from the Wellcome Sanger Institute, and Prof Madeline Lancaster at the MRC Laboratory of Molecular Biology (LMB), together with clinical partners at Cambridge University Hospitals and Royal Papworth Hospital.

It will be based at the CSCI’s facilities in the Jeffrey Cheah Biomedical Centre on the Cambridge Biomedical Campus, benefitting from close proximity to NHS partners, the LMB, AstraZeneca’s Discovery Centre, GSK’s Clinical Unit and other research and industry organisations.

Science minister Chris McDonald said: "Too many medicines that work in animals go on to fail in people. The Preclinical Translational Models Hub will allow scientists to test treatments on human tissue, grown from patients' own cells, so we can tell much earlier what will genuinely work.

“By opening that technology up to researchers, the NHS and companies across the country, we are bringing the end of animal testing closer while creating the high-skilled jobs and growth that benefit people in every postcode."

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