Cambridge to lead new £20m MRC Hub to transform development of new medicines using stem cells and AI

The Hub builds on the expertise of the Cambridge Stem Cell Institute, in particular around advanced human in vitro models, including organoids and other stem cell-derived systems. It will combine this with AI-powered approaches, bioengineering and clinical research to create more accurate and predictive ways of studying human disease and testing potential treatments before they reach patients.

The Hub will be led by Professor Matthias Zilbauer and Professor Bertie Göttgens from the Cambridge Stem Cell Institute (CSCI).

Professor Matthias Zilbauer, Director of the UK Pre-clinical Translational Models Hub and 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."

Working with hospitals, research institutes and industry partners across Cambridge and the UK, the researchers will establish a national network dedicated to developing, validating and accelerating the adoption of next-generation preclinical models. The Hub will enable researchers, clinicians and companies across the UK to access human-based platforms that better reflect human biology and improve the development of new therapies.

Professor Bertie Göttgens, Co-Director of the UK Pre-clinical Translational Models Hub and 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."

Every new medicine must undergo extensive testing for safety and effectiveness before it can reach patients. However, the process relies heavily on animal models, and many treatments that appear promising in animals ultimately fail when tested in humans. This can result in years of research, significant investment and potential treatments being lost before they reach patients.

Advanced human in vitro models and AI offer a new approach

By using human cells and tissues to recreate aspects of disease in the laboratory, researchers can study disease processes more accurately, identify potential treatments earlier and better predict how patients may respond to new therapies. These approaches include organoids - miniature, simplified versions of human tissues - alongside other engineered human systems and computational models.

While these technologies have already transformed many areas of biomedical research, their wider use in drug development requires robust validation, standardisation and benchmarking. Researchers, clinicians, regulators and industry partners need confidence that these models are reliable, reproducible and capable of improving decision-making in the development of new medicines.

The Pre-clinical Translational Models Hub will address this challenge by creating a national UK accelerator platform to develop promising human-based models into validated, scalable and industry-ready technologies. By integrating experimental models with AI and other computational approaches, the Hub will help establish a new generation of predictive tools for understanding disease and accelerating therapeutic discovery.

Cambridge's expertise in organoid research

Organoids and other advanced human models have become a major focus of stem cell biology since the field emerged in 2009, and CSCI has played a leading role in developing this area. Researchers across the Institute have contributed to advances in organoid technology, stem cell biology and disease modelling.

Professor Zilbauer, for example, has built a biobank of more than 1,000 patient-derived organoid lines over the past decade. Through his 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 and use these systems to understand disease mechanisms and test potential new treatments.

Professor Zilbauer said: "Patient-derived models have enormous potential to transform the way we understand disease and develop new medicines. 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."

To maximise the Hub's reach and impact, CSCI will work with Affiliate Group Leaders and Hub co-leads Dr Mathew Garnett and Dr Mo Lotfollahi from the Wellcome Sanger Institute, and Professor Madeline Lancaster at the MRC Laboratory of Molecular Biology, together with clinical partners at Cambridge University Hospitals and Royal Papworth Hospital.

Based at CSCI's facilities in the Jeffrey Cheah Biomedical Centre on the Cambridge Biomedical Campus, the Hub will benefit from close proximity to NHS partners, the MRC-LMB, AstraZeneca's Discovery Centre, GSK's Clinical Unit and other leading research and industry organisations. This unique environment will enable collaboration across disciplines and help accelerate the translation of scientific discoveries into real-world impact.

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."

Adapted from a news story by the Cambridge Stem Cell Institute