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

A new MRC Hub will create more targeted treatments for disease using advanced human in vitro models and AI

The Medical Research Council (MRC) has announced a £20 million investment in a new Pre-clinical Translational Models Hub, led by the Cambridge Stem Cell Institute (CSCI), which will transform the development and testing of new medicines by advancing the use of human-based models and artificial intelligence (AI).

The Hub will bring together expertise in advanced human in vitro models, including organoids and other stem cell-derived systems, AI-powered approaches, bioengineering and clinical research to create more accurate and predictive ways of studying human disease and evaluating potential treatments before they reach patients.

The Hub will be led by Professor Matthias Zilbauer, Group Leader at the Cambridge Stem Cell Institute and Honorary Consultant Paediatric Gastroenterologist at Cambridge University Hospitals, and Professor Bertie Göttgens, Director of the Cambridge Stem Cell Institute.

Working with hospitals, research institutes and industry partners across Cambridge and the UK, they 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.

Every new medicine must undergo extensive testing for safety and effectiveness before it can reach patients. However, the process still 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.

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 (IBD) 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 (MRC-LMB), together with clinical partners at Cambridge University Hospitals NHS Foundation Trust, Milner Therapeutics Institute, and Royal Papworth Hospital NHS Foundation Trust .

By combining expertise across stem cell biology, organoids, bioengineering, artificial intelligence, computational modelling and pharmaceutical collaboration, the Hub aims to become a national resource for academia, the NHS and industry, supporting the development of more effective and safer treatments for patients.

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.

Professor Göttgens, Co-Director of the UK Pre-clinical Translational Models Hub and Director of the Cambridge Stem Cell Institute, said:

“By combining expertise in stem cell biology, advanced human models and artificial intelligence, the UK Pre-clinical Translational Models Hub will accelerate the development of the next generation of predictive disease models. Working with our academic, NHS and industry partners, we aim to deliver better approaches for developing new treatments while reinforcing the UK’s global leadership in this field.”