A human focus for 21st century life sciences

03 - 04 February 2026 09:00 - 17:00 One Birdcage Walk Free Watch online

Science+ meeting organised by Professor Geoffrey J Pilkington, Ms Rebecca Ram and Ms Kathy Archibald on behalf of the Alliance for Human Relevant Science.

This meeting showcased the transformative value of innovative, human-focused biomedical technologies, which are providing invaluable insights into human diseases and could underpin the development and optimal use of new and repurposed medicines. It brought together researchers, clinicians, industries, regulators and others to discuss key challenges and how to surmount them to to accelerate their advantageous development and implementation. 

Image credit: iStock.com / sturti

Organisers

  • Geoff Pilkington

    Professor Geoffrey J. Pilkington

    Geoff Pilkington is an Emeritus Professor of Neuro-oncology, having held chairs at King's College, London and the University of Portsmouth, where he was Director of the Brain Tumour Research Centre which exclusively used human cells, tissues and biomaterials in their research. He is a Past President of the British Neuro-oncology Society and Honorary Treasurer, Executive Board Member and Scientific Board Member of the European Association of Neuro-oncology as well as being a Fellow of the Royal College of Pathologists and a Fellow of the Royal Society of Biology. His published work was largely focussed around brain tumour invasion, the blood brain barrier, the tumour microenvironment, brain tumour modelling and repurposed drugs.

  • Rebecca Ram

    Ms Rebecca Ram

    Rebecca Ram is an independent scientific consultant to the Safer Medicines Trust, working in the field of human-relevant science for almost two decades. Prior to this, she worked for a decade as a clinical data manager and programmer within phase 1-IV clinical trials for pharmaceutical and clinical research organisations(CROs). Rebecca has a Masters (MSc) in Toxicology (with Bioinformatics) and a BSc in Applied Biology. Her particular interest is in the field of clinical and scientific research and policy, including development and regulatory acceptance of advanced in-vitro and computational methods, improved use of clinical data and  'big data' analysis, as well as transition to human relevant approaches in fundamental research and disease modelling.  Rebecca has written or co-authored occasional research papers and has presented at webinars, universities and colleges. She has also served on review committees for conference abstracts, publications and early career research awards.

  • Kathy Archibald

    Mrs Kathy Archibald

    Kathy Archibald is the chair of Safer Medicines Trust. She graduated in genetics from Nottingham University and went on to work for Searle Pharmaceuticals and Genetics International (which became MediSense before acquisition by Abbott Laboratories). She then spent a decade working in conservation, including as a teacher-naturalist for the RSPB, followed by educational fundraising in schools for Action for Children. She founded Safer Medicines Trust in 2005, to confront the poor relevance of much pharmaceutical research (based on animals) to human medicine and its serious consequences for patient safety. The Trust has held conferences at the Royal Society and the House of Lords, initiated the Safety of Medicines Bill and published many peer-reviewed papers, book chapters and a popular science book, Rat Trap. Safer Medicines Trust was a founder member of the Alliance for Human Relevant Science, which helped establish the All-Party Parliamentary Group on Human Relevant Science in 2020.

Schedule

Chair

Michael Coleman

Professor Michael Coleman

Aston University, UK

09:00-09:05 Welcome by the Royal Society and lead organiser
Professor Geoffrey J.  Pilkington

Professor Geoffrey J. Pilkington

University of Portsmouth, UK

09:05-09:25 A 50-year career reflection on models of human disease in research and pre-clinical testing: where are we now?

During Professor Pilkington’s long career in neuro-oncology, he developed novel models for the biology and development of brain tumours and pre-clinical testing systems for drug efficacy, delivery, and mechanisms of therapeutic resistance.

In his early career, such models were developed in rats and mice, but he became aware that in addition to the ethical issues around this approach, such approaches, while of scientific interest, did not represent the situation in human neuro-oncology. Nor did they accurately represent other human neuropathologies, including dementias. He will allude to examples of where animal models were, and still are, imperfect.

Later in his career, he used in vitro approaches derived from human biopsy tissues which, although initially suffering from other flaws, due to poor design, were adapted and improved. These models became increasingly sophisticated and more akin to human brain pathologies. Ultimately, he established a brain tumour research centre which focussed on human biopsy-derived cells in in vitro models of the blood-brain barrier, brain tumour invasion/microenvironment and pre-clinical testing in 3D multiple cell type assays, providing a more accurate representation of human disease.

He will address the remaining flaws in both animal and ‘all-human’ modelling as well as how advanced human modelling is now ready to be exploited to determine the likelihood of well-designed clinical trials leading to successful outcomes for patients. He will close by suggesting that the orchestration of multiple new advanced methods will result in safer predictions for clinical trial outcomes, particularly in the testing of repurposed and re-formulated drugs.

Professor Geoffrey J.  Pilkington

Professor Geoffrey J. Pilkington

University of Portsmouth, UK

09:25-09:45 The new revolution in medicine: AI, imaging and the future of health care

The spreading of digital health innovation and personalized medicine together to the related regulatory shifts are the challenges of next future. The diffusion of AI worldwide appears to be a key point in the development of the most innovative devices allowing an easier and faster access to the cutting-edge technologies supporting the clinical practice and optimizing the management of health care related costs.

AI techniques concerning process mining in medical procedures will be the cornerstone in the balance between resources and clinical needs, improving the standard of care for patients. Generative AI creates new data and opportunities especially when applied to medical imaging also supporting research and improving the development of new technologies in medical imaging through data analysis. Radiomic features may be used to allow an earlier disease detection, improving prognostic assessment as well as a better treatment strategy definition. The future of precision medicine relies on imaging, achieved by the implementation of radiomics technologies, and theranostics, with the same agent able to diagnose and treat simultaneously. This will be the best example individual personalized medicine.

Generative AI has also demonstrated the power to increase the diagnostic performance, improving the accuracy and precision, potentially reducing costs and risks for population. Application of generative AI regulation changes is often slower than innovation. Anyway, AI requires legislative changes, and one of the most significant challenges will be managing technological progress as fast as it is need ensuring the optimal regulatory context while preserving every opportunity for progress within the healthcare system.

Professor Orazio Schillaci

Professor Orazio Schillaci

Italian Minister of Health

09:45-10:00 Discussion
10:00-10:20 Break
10:20-10:40 HNRNPD: Guardian of the (beta cell) galaxy

Cellular stressors are inducers of cell identity change. In the pancreatic islet, factors such as high glucose and dysregulated lipid can cause cellular transdifferentiation events. This has been observed in human beta cells in vitro and in islets of people with diabetes.

Using a completely humanised system, we investigated the causes of pancreatic cell identity change by defining global gene expression patterns in normal and transdifferentiated cells after exposure to stress. We then tested the most dysregulated genes for causality using siRNA gene knockdown and hormone staining. We then investigated the impact of splicing-associated changes in the binding and expression of downstream targets. Finally, we explored the potential for reversal of cell identity changes by restoration of euglycaemia or manipulation of candidate isoform levels.

We identified the heterogeneous ribonucleoprotein D (HNRNPD) gene as a potent protector of beta cell identity in the face of cellular stress. Cellular stressors caused a change in the splicing patterns of HNRNPD, with consequent disruption of the downstream beta cell development and function gene expression programs. We were able to demonstrate reversal of transdifferentiation events by removal of stress or manipulation of HNRNPD isoforms.

We have identified HNRNPD as a key factor involved in the protection of beta cell identity. HNRNPD splicing changes induced by disrupted metabolism cause a reversible switch from a beta cell to delta cell profile. The nature of the change is human-specific and underlines the importance of using human-relevant experimental systems to identify human-relevant disease processes. These findings may represent a potential future point of traction for protection of beta cell mass in face of diabetes.

Professor Lorna Harries

Professor Lorna Harries

Exeter University, UK

10:40-11:00 Drug-induced liver injury: Genetic architecture to precision medicine

Liver is one of the most common organ toxicities observed during pre-clinical development, yet the signals of hepatotoxicity found in animal models are deemed unreliable. A systematic review demonstrated liver and immune related adverse events in combination accounted for 30% of the market withdrawal of medications. Among 62 drugs approved by FDA in 2021, 58% have been shown to have hepatotoxicity in clinical trials of listed in the official label. Host genetic factors are important in determining the susceptibility to idiosyncratic drug-induced liver injury (DILI) which is the most common cause for acute liver injury and the second 3rd in causes of jaundice presenting to secondary care hospitals. Through large international collaborations, and involvement of robustly evaluated patients with DILI, we have identified several human leucocyte antigen (HLA) and non-HLA genetic risk factors increasing susceptibility to DILI. Overlapping HLA alleles determine risk of DILI due to more than 20 drugs. Genetic risk scores using multiple genotypes have been developed to assist the diagnosis and causality assessment of DILI due to specific drugs. Genetic architecture determines the DILI potential of several drugs in organoids derived from primary hepatocytes or hepatocyte like cells derived from inducible pluripotent stem cells. This ‘polygenicity in dish’ concept will be the path to developing human relevant in-vitro models for the pre-clinical evaluations during the drug development. Recent cluster randomised controlled implementation trial demonstrated that a platform with 50 variants related to 12 genes contributing to pharmacogenomic alteration in 44 drugs can reduce adverse drug reaction by 30%. This has set a path for embedding precision medicine in the community.

Professor Guruprasad Padur Aithal

Professor Guruprasad Padur Aithal

University of Nottingham, UK

11:00-11:15 Discussion
11:15-12:15 Roundtable discussion: Why should medicine be human-focused?
Dr David Bunton

Dr David Bunton

REPROCELL Europe Ltd, UK

Professor John Greenman

Professor John Greenman

University of Hull, UK

Professor Christine Mummery

Professor Christine Mummery

Leiden University Medical Center, The Netherlands

Professor Denis Noble CBE FMedSci FRS

Professor Denis Noble CBE FMedSci FRS

University of Oxford, UK

Professor Helen Maddock

Professor Helen Maddock

InoCardia, UK

Dr Mona Boyé

Dr Mona Boyé

Ksilink, France

Chair

Malcolm Wilkinson

Dr J Malcolm Wilkinson

Sheffield University, UK

13:15-13:20 Chair's introduction
Dr J Malcolm Wilkinson

Dr J Malcolm Wilkinson

Sheffield University, UK

13:20-13:35 Optional ornament or crucial code? The role of modelling and simulation in human safety assessment

Human safety assessment in drug development is undergoing significant change, driven by high attrition rates, ethical concerns about animal testing, and new global regulations encouraging alternative methods. Quantitative Systems Toxicology (QST), a key technology within New Approach Methodologies (NAM), uses mathematical modelling and experimental data to predict toxicological effects more mechanistically than traditional models. QST provides mechanistic, human-relevant evidence vital for decision-making, risk assessment, and regulatory submissions.

International regulators increasingly recognise QST, as seen in recent guidelines from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use on General Principles for Model-Informed Drug Development (ICH-M15) and updates like the FDA Modernization Act 2.0. QST offers mechanistic insights into toxicity, improves translational predictions of human responses, and supports efforts to reduce animal testing, aligning with the 3Rs principle (reduction, refinement, replacement). Case studies in organ-specific toxicities, and advanced therapies show QST’s impact on safer drug design and regulatory approval. As drug safety continues to evolve, QST is set to become a crucial, rather than optional, part of the drug development and safety assessment, advancing scientific and ethical standards in modern drug development.

Dr Ciarán Fisher

Dr Ciarán Fisher

GSK, UK

13:35-13:50 Human-based in silico medicine for drug development

Precision Medicine aims at providing the most accurate diagnosis and best treatments for each patient. Whereas this has primarily been genomic-centred so far, there is now a wide recognition of the need to consider a wide spectrum of lifestyle, environment, and biology conditions. Characterising such diversity of factors requires large quantity and quality of patients’ datasets, and at the same time, innovative approaches for their analysis, drawing on the increasing power of computers and algorithms. In this presentation, I will illustrate progress towards the vision of the Digital Twin and In Silico Trials for therapy testing, through human-based modelling and simulation for prediction medicine, therapy development, and the 3Rs of animals in research. These concepts are broadly applicable through all areas of medicine, and I will describe our own progress in cardiology and drug safety assessment. I will aim to demonstrate how combined computational approaches, including modelling and simulation and machine learning, can boost the capacity for diagnosis and prognosis, as well as testing of future treatments.

Professor Blanca Rodriguez

Professor Blanca Rodriguez

University of Oxford, UK

13:50-14:05 Enhancing preclinical risk assessment with computational tools

If impactful safety issues are first detected during the pre-IND GLP in vivo safety studies, this is far too late.  The chemistry is already ‘locked-in’ to the candidate drug and there will have been significant investment in terms of compound synthesis of sufficient quantities for Phase I clinical trials and the cost of the tox IND package, not to mention the resource costs of getting to that point.  To minimise the risk of this, toxicologists and safety pharmacologists generate in vitro safety data long before then.  However, this only provides a patchy coverage of the entire spectrum of potential toxicities.  There is a clear opportunity to (a) construct QSAR/QSTR models to flag-up various safety issues even before compound synthesis and then, once compounds are synthesised, (b) to apply computational safety tools to improve interpretation of in vitro readouts.  By focusing on the most common and impactful preclinical safety issues (hepatotoxicity; QT prolongation; off-target safety), the discovery small molecule pipeline can be significantly de-risked during lead optimization.  Going forwards, to accord with the FDA’s 2025 Roadmap to Reducing Animal Testing in Preclinical Safety Studies and the UK Government’s 2025 Replacing Animals in Science Strategy, and replace in vivo safety studies with New Approach Methodologies (NAMs), will require a coordinated involvement of both in vitro and in silico technologies.  Some of these will have to be promoted from their current front-loading role to a frontline one, as well as the development of new ones.

Dr Will Redfern

Dr Will Redfern

Certara, UK

14:05-14:20 Discussion
14:20-14:45 Break
14:45-15:00 From promise to practice: enabling human relevant, pharma-ready life sciences through policy, regulation, standards, and platforms

Human relevant technologies such as New Approach Methodologies (NAMs), microphysiological systems (MPS), and organ-on-chip platforms are increasingly recognised as essential components of modern biomedical research and therapeutic development. Their growing importance reflects advances in experimental science, alongside supportive shifts in regulatory thinking, policy frameworks, AI and the development of shared standards that together are reshaping expectations of evidence and relevance.

Recent policy and regulatory developments highlight this changing landscape. In the United States, the FDA Modernization Acts have moved regulatory guidance away from prescriptive animal testing requirements toward broader acceptance of nonclinical evidence, explicitly enabling the use of human relevant and computational approaches. In the UK the national strategy for alternative methods similarly emphasise innovation grounded in human biology, while seeking to accelerate validation, confidence, and uptake across research and development. These signals are reinforced by increasing attention to standards, qualification pathways, and cross sector alignment.

At the same time, it is clear that human relevant platforms will not address every scientific or regulatory challenge, nor will they replace all animal models wholesale in the near term. NAMs, MPS, and organ on chip technologies are particularly powerful when applied to well defined questions, such as human specific mechanisms, integrated multi cellular and some system level functions, and aspects of safety assessment. Supporting technologies, including data driven and AI enabled analytical tools, play an important enabling role, helping to integrate evidence, manage complexity, and support decision making.

This talk will take a strategic, forward-looking view of how policy, technology platforms, standards, and enabling analytics are supporting the transition from promise to practice in human relevant life sciences. It will highlight where momentum is building, where challenges remain, and how the next decade could see these approaches more effectively embedded to support more predictive and efficient drug discovery and development, and ultimately improve outcomes for human health.

Dr Dharaminder Singh

Dr Dharaminder Singh

Formerly CN Bio, UK

15:00-15:20 Modelling health and disease on a chip: the rapidly evolving field of micro-physiological systems

Animal models, and mice especially, have proven critical in developing an understanding of human disease, but they are not without their limitations. Mice are short-lived relative to humans, their metabolism differs in significant ways and they genetics differs significantly enough that some human diseases simply cannot be meaningfully modelled. These limitations and the growing push to reduce the use of experimental animals has driven the rise of microphysiological systems (MPS), also called complex in vitro models (CIVM) and more colloquially, organ-on-chip. The vasculature is a multifunctional organ critical for life. In addition to facilitating the distribution of oxygen and nutrients across tissues of the body, blood vessels play a key role in the active transport of immune cells, metabolites, and therapeutics, and in the regulation of blood pressure and the maintenance of normal body temperature. MPS platforms that include perfused human vasculature thus offer a more physiologic model system in which to further advance our understanding of human disease. We have developed the Vascularized Micro-Organ platform (VMO), which incorporates a microfluidic artery and vein connected by a spontaneously assembled microvascular network. A blood substitute is driven through the vasculature by gravity and supports surrounding tissue, which can either be a generic stroma, organ-specific, or a tumour. Based on this platform we have developed models for several diseases including cancer, diabetes and vascular malformations. We are using these to investigate biological mechanisms underlying disease, and to identify and validate therapeutic targets.

Professor Christopher Hughes

Professor Christopher Hughes

University of California, Irvine, US

15:20-15:30 Discussion
15:30-16:30 Roundtable discussion: Is the government correct that the use of animals will be essential for some time to come?
Professor Richard Luxton

Professor Richard Luxton

University of the West of England, UK

Dr Gerry Kenna

Dr Gerry Kenna

Kenna Consulting, UK

Dr Katya (Tsaioun) Hélin

Dr Katya (Tsaioun) Hélin

Akttyva Therapeutics, US

Professor Michael Coleman

Professor Michael Coleman

Aston University, UK

Professor Lorna Harries

Professor Lorna Harries

Exeter University, UK

The Rt Hon George Eustice

The Rt Hon George Eustice

Former Secretary of State for Environment, Food and Rural Affairs, UK

16:30-16:50 Flash talks x 4
16:50-17:00 Day 1 closing remarks
Professor Geoffrey J.  Pilkington

Professor Geoffrey J. Pilkington

University of Portsmouth, UK

17:00-18:15 Poster session

Chair

John Greenman

Professor John Greenman

University of Hull, UK

09:00-09:05 Opening remarks and Chair's introduction
Professor John Greenman

Professor John Greenman

University of Hull, UK

09:05-09:25 Drug safety, pharmacogenomics and personalised medicine (online)

Adverse drug reactions (ADRs) remain a major, perhaps increasing, burden on healthcare systems. The burden often falls on the most vulnerable including the youngest and oldest (with multiple diseases) who are not usually included in clinical studies. Our latest data in the elderly with multimorbidity and polypharmacy indicates that 16.5% of admissions to our hospitals are due to ADRs, costing the NHS in England £2.21 billion per year.

Although animal studies allow us to understand the pharmacokinetics of a drug in vivo, and potentially predict type A dose-dependent reactions, they are not usually designed to identify individual susceptibility factors. For example, it is not possible to undertake non-clinical studies that replicate the complexity encountered in a patient with multimorbidity and polypharmacy. This may however be possible by using modelling approaches but again limited because by the fact that few studies involve the elderly who are living with multiple long-term conditions. For type B (bizarre, unpredictable) reactions there are no good animal models to predict individual susceptibility. Because of the dearth of relevant studies during drug development, the identification of individual susceptibility to ADRs usually occurs during the post-marketing phase in real-world settings.

Pharmacogenomics is the study of how genetic variation in individuals determines the response to a drug (both efficacy and safety). Pharmacogenomics represents one component of (a) factors that determine variability in drug responses; and (b) the overall field of personalized medicine. The presentation will discuss examples of how pharmacogenomics is being used in clinical practice to reduce ADRs and improve patient outcomes, and how this might be implemented in the future to benefit the whole population.

Sir Munir Pirmohamed

Sir Munir Pirmohamed

University of Liverpool, UK

09:25-09:45 Cardiovascular diseases and drugs: Where are we with hiPSC models?

Derivation of cardiovascular cell types from human pluripotent stem cells (hPSCs) generated from patient tissues or introducing targeted mutations into healthy control hPSCs is an area of growing interest as a platform for disease modelling, drug discovery and (cardio)toxicity. Our lab has been investigating microtissue solutions in which cardiomyocytes and cardiac vascular and stromal cells are present. The presence of cardiac stromal cells promotes cardiomyocyte maturation. By combining cardiac microtissues with new methods for functional phenotyping, we have been able to quantify the outcomes of drug and disease mutation responses in situ in high throughput. The use of isogenic pairs of hiPSC lines with and without mutations has proven very important since variability between “healthy control” hiPSC lines is often greater than the difference between diseased cells and its isogenic control. Examples of studies investigating disease and drug responses in cardiac microtissues will be shown. These approaches have allowed us to identify which cell types in the heart are responsible for the disease phenotypes observed in vitro indicating which cells we might target for therapy. Moreover, we have been successful in automating the production of healthy and disease cardiac microtissues such that they can be used for high throughput drug screens. These complex cell systems are paving the way towards better understanding of disease mechanisms and drug discovery.

Professor Christine Mummery

Professor Christine Mummery

Leiden University Medical Center, The Netherlands

09:45-10:00 Discussion
10:00-10:20 Break
10:20-10:40 3D cell culture models and their role in enhancing genotoxicity testing approaches for nano- and advanced-materials

Standard in vitro genotoxicity testing approaches have limitations for testing nanomaterials (NMs) and in 2013, an OECD expert panel concluded it was necessary to adapt the in vitro mammalian cell micronucleus test (OECD TG487) to facilitate evaluation of manufactured NMs. An OECD project was initiated, aimed at developing a new OECD Guidance Document detailing the necessary steps to adapt OECD TG487 for NMs, which was subsequently published in Sept 2022 (Series on Testing and Assessment No. 359; ENV/CBC/MONO(2022)15). Whilst ongoing efforts have focused on adapting existing OECD TGs for NMs testing, limitations remain as existing in vitro approaches lack physiological relevance, often do not consider long-term exposure effects, and do not cover all mechanisms of action underpinning genotoxicity. New approach methodologies (NAMs) provide the opportunity to overcome these issues and there have been substantial developments in this area over recent years. For example, co-culture models incorporating epithelial and macrophage cells can detect secondary genotoxicity, a key nanomaterial mode of action only detected in vivo. Additionally, 3D liver spheroids exhibit higher metabolic capacity, which is important for identifying pro-carcinogens. These models also support longer-term, repeated dosing, which is more representative of nanomaterial human exposure. The broad field of NAMs to better support regulatory decision making is therefore gaining momentum, with a variety of promising technologies emerging.

Professor Shareen H Doak

Professor Shareen H Doak

Medicines and Healthcare products Regulatory Agency (MHRA), UK

10:40-11:00 Which contexts of use arise from animal studies for new approach methods development? How does this change impact staff and infrastructure?

It is critical to understand the in vivo animal test (IAT) when developing and accelerating adoption of New Approach Methodologies (NAMs). The specialist animal staff understand the strengths and weaknesses of their IATs, and their knowledge and experience are needed when developing and implementing NAMs. Each IAT performed results in many outputs (eg liver weight changes, clinical signs, biomarkers, metabolites formed etc) that are used to decide whether there is an adverse event or not. Each output can be converted into a context of use (CoU), with adverse outcome pathways devised and used to create NAMs (eg in vitro or computational tests). Many NAMs, alone or as batteries, are required to replicate all animal tests endpoints. The same process can be followed to replicate the CoUs in human disease. Both IATs and human NAMs should predict the human disease to be of value in a risk assessment or submission in a regulatory package. How does this impact on the staff working on these tests and the infrastructure? Whilst NAMs are phased in, those staff working on the animal tests can be trained in cell husbandry, dosing, and sample collection. This is no different to when they are retrained to work with different species. The technologies and knowledge used by support services, eg pathology, immunology and bioanalysis, are similar for NAMs and minimum retraining is envisaged. When repurposing a vivarium to a tissue culture lab, smaller rooms are needed, but temperature and humidity control is essential. NAM development needs stakeholders with animal test knowledge to accurately develop CoUs for NAMs development.

Dr Clive Roper

Dr Clive Roper

Roper Toxicology Consulting Limited, UK

11:00-11:15 Discussion
11:15-12:15 Panel discussion: How would a transition to human-relevant methods make medicines safer?
Dr Stella Cochrane

Dr Stella Cochrane

Unilever SERS (Safety, Environmental and Regulatory Science), UK

Professor Chris Molloy

Professor Chris Molloy

Medicines Discovery Catapult, UK

Professor Helen Maddock

Professor Helen Maddock

InoCardia, UK

Professor Christopher Hughes

Professor Christopher Hughes

University of California, Irvine, US

Professor Blanca Rodriguez

Professor Blanca Rodriguez

University of Oxford, UK

Professor Geoffrey J.  Pilkington

Professor Geoffrey J. Pilkington

University of Portsmouth, UK

Chair

Katya Helin

Dr Katya (Tsaioun) Hélin

Akttyva Therapeutics, US

13:15-13:20 Chair's introduction
Dr Katya (Tsaioun) Hélin

Dr Katya (Tsaioun) Hélin

Akttyva Therapeutics, US

13:20-13:40 Why we need social sciences to understand our behaviour and make faster progress together

As a newly graduated veterinarian in 1986, I aimed to improve animal welfare and scientific quality to enhance the translation of animal research to humans. At the time, I believed animal studies were necessary and evidence-based. Early in my career, I focused on implementing the 3Rs, particularly Reduction and Refinement. Over time, however, I realized that the 3Rs alone were insufficient. Systematic reviews proved more effective, as they directly supported 3R implementation by e.g. identifying unnecessary duplication. These reviews also revealed the poor quality of animal study publications and the lack of (predictors for) successful translation to humans.

This led me to shift away from animal studies toward more human-relevant science, especially given advances in New Approach Methods (NAMs) such as organoids, AI, and better use of human-based data. Together with historians, I analysed animal testing requirements in legislation and found they were largely based on crisis-driven decisions rather than solid scientific evidence. The COVID-19 pandemic further demonstrated that development timelines for vaccines could be drastically shortened through increased use of NAMs, reduced animal testing, use of existing data, and earlier clinical trials.

In the Netherlands, I joined the government-initiated Transition to Animal-Free Innovations program, applying transition /social sciences to accelerate this shift. This interdisciplinary approach brings together multiple disciplines in academia, industry, policymakers, and regulators. Supported by dedicated funding, I became co-chair of the SAFE consortium (Safety Assessment through animal-Free Evolution), which studies barriers to regulatory adoption of animal-free methods and how to accelerate progress toward more human-relevant science.

Professor Merel Ritskes-Hoitinga

Professor Merel Ritskes-Hoitinga

Utrecht University, The Netherlands

13:40-14:00 How much top-down policy in transitions beyond animal experimentation?

In recent years, several national governments, the European Commission, and several US government agencies have made efforts to strategize for a transition beyond animal experimentation. In debates about such strategies, the role of top-down policies is controversial. While animal advocates have called on governments to commit to specific reduction goals, or even to put an expiry date on some or all forms of animal experimentation, groups representing science and industry have warned that innovation cannot be planned and government should not try to steer research top-down. Synthesizing arguments on either side of this debate, this talk will suggest that policy mixes combining top-down and bottom-up approaches – drawing on frameworks such as Transformative Governance, Transition Management, and Mission-Oriented Research and Innovation – can retain most of what transition strategy advocates seek while avoiding most of the risks highlighted by their opponents.

Dr Nico Müller

Dr Nico Müller

University of Basel, Switzerland

14:00-14:20 Ksilink – a patient-based phenotypic drug discovery engine

Late-stage clinical trial failures in drug development are often driven by poor efficacy, largely due to inappropriate animal models that fail to capture human disease complexity. Ksilink addresses this challenge through a patient-based phenotypic drug discovery engine, leveraging human cellular systems derived from patients for high-throughput screening. By automating complex human disease models through AI-driven image analysis, this platform enables physiologically relevant insights without relying on animal models. It supports screening of chemical and genetic perturbators, target deconvolution, and mechanism-of-action profiling directly in human cells. This approach not only accelerates discovery but also improves translational predictability, reducing ethical concerns and enhancing the likelihood of clinical success.

Dr Mona Boyé

Dr Mona Boyé

Ksilink, France

14:20-14:30 Discussion
14:30-14:45 Break
14:45-15:15 Modernization at the US FDA

Learn about key modernisation initiatives and top priorities for 2026 at the new FDA.

Dr Martin Makary

Dr Martin Makary

Food and Drug Administration, US

15:15-15:35 The role of biological computational models in human-oriented medical research

My research has focussed on using experimental data from animal and human research to develop mathematical models of the heart, starting in 1960 with the first successful model explaining how heart rhythm arises from interactions between the cell membrane potential and the proteins carrying ionic currents. Those models, now including models specifically based on human cardiac cells, have been used extensively in the pharmaceutical industry to test for possible arrhythmic side-effects. They may also be used to clarify the difference between association scores in genome-wide association studies and physiological causation. In the case of the heart this can be as large as 85% causation with only a 10-15% association score (Noble, 2025). This difference is attributable to the presence of robust back-up processes, and would explain why the great majority of association scores are too small to be useful in predicting disease states. That fact is confirmed by the demonstration that the polygenic scores do not reliably predict either cancer or cardiovascular disease (Hingorani et al 2023). The outcome is what I call a gene-centric impasse: the failure of genomics to result in successful diagnoses and cures. I will conclude by showing how physiological modelling of causation may solve the gene-centric impasse.

Hingorani, A.D., Gratton, J., Finan, C., et al 2023. BMJMedicine. 2:e000554. doi:10.1136/ bmjmed-2023-000554
Noble, D. 2025. The Pacemaker Channels of the Heart: from reductionism to systems biology. World Scientific Press.

Professor Denis Noble CBE FMedSci FRS

Professor Denis Noble CBE FMedSci FRS

University of Oxford, UK

15:35-15:45 Discussion
15:45-16:45 Panel discussion: How can we drive a transition to human-relevant science in academic research as well as preclinical testing?
Professor Merel Ritskes-Hoitinga

Professor Merel Ritskes-Hoitinga

Utrecht University, The Netherlands

Dr J Malcolm Wilkinson

Dr J Malcolm Wilkinson

Sheffield University, UK

Professor Paul L Carmichael

Professor Paul L Carmichael

Unilever, UK

Dr Nico Müller

Dr Nico Müller

University of Basel, Switzerland

Dr Mona Boyé

Dr Mona Boyé

Ksilink, France

Steve Race

Steve Race

Labour MP for Exeter, UK

16:45-17:00 Reflections, next steps and concluding remarks from co-organisers
Professor Geoffrey J.  Pilkington

Professor Geoffrey J. Pilkington

University of Portsmouth, UK

Ms Rebecca Ram

Ms Rebecca Ram

Safer Medicines Trust, UK

Mrs Kathy Archibald

Mrs Kathy Archibald

Safer Medicines Trust, UK