Mohamed Hassanein, Director of Translational Medicine, Pfizer, and Daniel Strasser, Senior Director and head of Translational Biomarker group, Idorsia led an exciting discussion on the significance of applying translational research to precision medicine. They both presented examples of how scientists can use biomarkers to drive drug development and tailor treatments to specific patient populations.
To get the ball rolling, Hassanein highlighted several case studies demonstrating how translational medicine is changing the treatment paradigm for certain inflammatory diseases. Inflammatory bowel disease refers to a group of chronic inflammatory diseases that impact the gastrointestinal tract, particularly the large colon. The known cause has not been determined but researchers believe it is a result of a combination of inflammatory, genetic, and environmental factors.
Hassanein partially attributed the complexity and heterogeneity of IBD to the low response rates to current treatments and the need for novel therapeutics targeting specific pathways. Currently, there are around 3 mechanisms of action for targeted therapies for IBD. The most well-known approach targets the inflammatory cytokine by neutralising antibodies such as TNF alpha. Other approaches inhibit IL23 and IL12 to reduce inflammation and inhibit the trafficking of pathogenic immune cells to the gut by blocking its receptor that binds to endothelial cells lining the colon. Regardless of these various treatment options, the patient response rate is very low (15 -25%).
So, to combat this unmet need, Hassanein stressed the need for biomarker-driven strategies to improve outcomes, citing tools like multiomics, gut microbiome profiling, and AI integration. He presented case studies showing how biomarkers such as IL-22 levels and plasma cell infiltration can predict treatment response, advocating for a shift from symptom-based to biomarker-guided therapies.
Strasser expanded on the challenges and opportunities of applying precision medicine. Given the complexity and heterogeneity of rare diseases and cancers, one must consider multi-faceted factors and the different disease-driving pathways, and understanding these factors requires multiple measurements and assessments. However, early integration of biomarker strategies in drug development and human-relevant models will be crucial to advancing precision medicine.
Diseases like IBD and rheumatoid share overlapping pathways and disease phases which is likely to complicate treatment options. Yet 2025 marks an exciting time, with the boom of spatial transcriptomics, 3D imaging, and AI it is hoped that capturing disease heterogeneity and guiding clinical trial design will be more straightforward than ever.
Strasser and Hassanein concluded that while precision medicine has seen success in oncology and rare diseases, its broader application requires integrating diverse data types, embracing AI, and fostering cross-sector partnerships. They underscored that although challenges remain, the evolving toolkit of translational research holds great promise for transforming patient care across complex diseases.