On 7 February 2026, EATRIS brought together 114 registered participants for a highly engaging Digital Symposium on New Approach Methodologies (NAMs) to Accelerate Medicines Development and Repurposing. Hosted by the EATRIS Small Molecules Platform, the event highlighted how human-relevant, translationally robust approaches are reshaping drug discovery across therapeutic areas.
From innovation to implementation
In their introductory keynotes, Prof. Daniela Salvatori (CSO, Ombion) and Dr. Sílvia Almeida (CSO, AccelBio) framed NAMs as central to Europe’s transition towards more predictive, human-relevant biomedical science. They emphasised various strategic drivers: improving translational predictivity, reducing reliance on animal models, quality and reproducibility through automation, and strengthening Europe’s regulatory leadership.
It was underlined that this is a “critical regulatory and policy moment”, with rapid growth in NAM research fuelled by European funding and innovation programmes. Importantly, EATRIS and its members were positioned as connectors — providing infrastructure for harmonisation, validation and cross-border collaboration to move NAMs from promising innovation to regulatory-grade practice.
The message was clear: success will depend not only on scientific excellence, but on coordinated validation, stakeholder alignment and regulatory readiness.
Translational NAMs in action
The programme showcased concrete examples of NAMs delivering mechanistic insight and actionable translational data:
- Advanced hiPSC-derived kidney organoids were shown to recapitulate nephron structures and functional readouts, including uptake, cyst formation and long-term culture. These models demonstrated measurable drug-induced toxicity responses, supporting their use in preclinical safety decision-making.
- Drug repurposing, using patient-derived iPSCs and organoids, identified phosphodiesterase-5 inhibitors as candidate therapies for Leigh syndrome. Sildenafil corrected mitochondrial and neurodevelopmental defects in vitro and has progressed to EMA Orphan Drug Designation and a forthcoming EU clinical trial.
- A translational framework integrating imaging, circulating biomarkers and clinical parameters identified blood–brain barrier dysfunction as a mediator of early infarct growth with good predictive performance supporting biomarker-driven patient stratification and therapeutic targeting.
- Additional contributions demonstrated organotypic cultures (tumour on a chip), microphysiological systems, multi-omics-guided functional screening, digital twin models and AI-driven drug combination tools — all reinforcing the shift towards human-based, mechanism-anchored development pathways.
Regulatory science and the European perspective
In the second keynote, Prof. Maurice Whelan (Head of Unit, Joint Research Centre) provided a regulatory science lens on NAM implementation. His perspective underscored the importance of robust validation, standardisation and evidence frameworks to enable regulatory uptake and invited the community to engage with JRC to jointly progress NAMs as a field of research.
Building on the scientific case presented throughout the day, the discussion reinforced that regulatory confidence depends on transparent performance evaluation, defined contexts of use, and cross-sector collaboration. The alignment between scientific innovation and policy evolution emerged as a central theme — positioning Europe to lead in the global transition towards animal-free, human-relevant testing strategies.
From promise to practice
A recurring message throughout the symposium was that NAMs are no longer conceptual alternatives — they are increasingly decision-informing tools. Whether enabling biomarker-guided stratification in stroke, identifying repurposed treatments for rare mitochondrial disease, or predicting organ-specific toxicity using advanced organoids, NAMs are directly contributing to de-risking development and accelerating translation.
With strong attendance, vibrant discussion and concrete translational case studies, the symposium demonstrated the growing maturity of the NAM field — and the pivotal role of EATRIS in connecting infrastructure, expertise and regulatory dialogue across Europe.
- Download the abstract book from the digital symposium.
- Learn more about the EATRIS Small Molecules Platform.
