Scientists at Puerta de Hierro University Hospital have successfully tested the first drug to improve cardiac function in cardiac amyloidosis, an incurable and often fatal disease. The research was published in the journal NEJM.
With amyloidosis of the heart, fibrils (threads) of amyloid proteins accumulate in it. This leads to thickening of the heart walls, increasing their stiffness, and heart failure. The prognosis is poor, and without supportive care, patients die an average of three years after diagnosis. The only way to restore the function of the heart is its transplant.
Preliminary results from a study involving 40 patients from France, the Netherlands, Germany and Spain showed that the new antibody-based drug was safe and reduced the amount of amyloid protein in the heart. The drug was administered intravenously to patients in gradually increasing monthly doses over a 12-month period.
“Patients who received higher doses of antibodies appeared to have a greater reduction in cardiac amyloid deposits and a greater improvement in a number of cardiac parameters,” the scientists wrote. Said.
The new drug, developed by the Swiss company Neurimmune, is an antibody that binds to the amyloid protein transthyretin, preventing it from accumulating in the heart. The antibody was isolated from immune B cells from healthy elderly people.
Cardiac amyloidosis can be caused by genetic mutations or aging. While currently available treatments are effective at preventing further amyloid fibrils from accumulating and slowing disease progression, they do not remove amyloid protein that has already accumulated in the heart.