Professor Marianna Fontana

Talk title: The future of ATTR management

Amyloid cardiomyopathy, particularly transthyretin (ATTR) amyloidosis, has undergone a profound therapeutic transformation over the past decade. Initial strategies focused on reducing the production of misfolded transthyretin through gene silencers and stabilisers, effectively slowing disease progression and improving outcomes. However, these approaches do not address the substantial burden of pre-existing amyloid deposits within the myocardium, which continue to drive cardiac dysfunction, particularly in advanced disease.

Emerging therapies now aim to enhance amyloid clearance, representing a complementary and potentially transformative approach. These agents are designed to target and remove fibrillar deposits, leading to a distinct pattern of cardiac remodelling characterised by structural changes and improved haemodynamic function. This shift has also prompted a re-evaluation of how treatment response is assessed, moving beyond traditional global metrics towards integrated measures that more accurately reflect myocardial substrate and function.

This talk will explore the evolving understanding of disease mechanisms in ATTR cardiomyopathy, from amyloid production to clearance, and discuss how these insights are shaping a more precise, mechanism-driven therapeutic paradigm. Ultimately, combining suppression of amyloid formation with active removal of existing deposits may offer the greatest opportunity to modify disease trajectory and improve patient outcomes.

Biography:

Marianna Fontana is Professor of Cardiology and Honorary Consultant Cardiologist at the Royal Free Hospital, University College London, Director of the UCL Cardiac MRI unit and Deputy Head and clinical lead of the Center for Amyloidosis, National Amyloid Center. Her major clinical and research interests are in the delivery of efficient and effective care for patients with amyloidosis, with a particular focus on new technologies: imaging and drugs. She is the recipient of numerous awards, including the Michael Davies Early Career Award in 2021, the Outstanding Contribution to Research Award RFH 2024, the AHA Visiting Professorship Award for Brigham and Women Hospital Harvard Medical School in 2024. She is widely published in the field of amyloidosis.

Association:

Marianna Fontana | About | University College London

Host:

Professor Mauro Giacca