Researchers at Puerta de Hierro University Hospital have announced promising progress in treating cardiac amyloidosis with the first drug shown to improve heart function in this condition. The findings were detailed in a major medical journal, NEJM.
Cardiac amyloidosis occurs when amyloid proteins assemble into fibrils that deposit in the heart. This buildup thickens the heart walls, stiffens the chamber walls, and promotes heart failure. The outlook for affected individuals is often poor, and without supportive care, survival following diagnosis typically ranges around three years. At present, the main intervention to restore heart function is transplantation, which remains a complex and high-risk option for many patients.
In a preliminary study involving 40 participants across France, the Netherlands, Germany, and Spain, researchers evaluated a novel antibody-based therapy. The treatment proved to be safe and appeared to reduce the burden of amyloid protein in the heart. The regimen involved intravenous administration with gradually escalating monthly doses over a full year.
According to the investigators, patients who received higher antibody doses showed a greater reduction in cardiac amyloid deposits and experienced improvements across several cardiac measurements, suggesting dose-responsive benefits and meaningful tissue-level change (study report, NEJM).
The drug was developed by a Swiss biotechnology company, and it targets the transthyretin protein, a key culprit in certain forms of amyloidosis. By binding to transthyretin, the antibody prevents the protein from accumulating in heart tissue, thereby limiting further damage and enabling a potential reversal of some functional impairments. The antibody was derived from immune B cells sourced from healthy older adults, a strategy designed to harness naturally occurring antibody capabilities.
Cardiac amyloidosis can arise from inherited genetic mutations or from aging-related changes. Current therapies, while effective at slowing the progression and preventing additional amyloid buildup, do not remove amyloid protein that already resides in the heart. As a result, researchers continue to pursue strategies that can both halt progression and promote clearance of existing deposits, aiming to improve symptoms, cardiac function, and long-term outcomes for patients.