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Transthyretin-related cardiac amyloidosis is a degenerative disease defined by the accumulation of amyloid protein fibrils in the heart. Amyloid fibril deposition thickens and stiffens the heart walls, causing stiff-heart syndrome. The buildup of amyloid fibrils causes heart failure, as well as fluid retention, tiredness, and arrhythmias. The condition can be caused by genetic abnormalities or by aging. The prognosis is bleak, with untreated patients living an average of only three years.
The findings of a study published in The New England Journal of Medicine (NEJM) promise to drastically change the prospects of people with this disease. Dr. Pablo Garcia-Pava, who heads the Inherited Cardiac Diseases Section at Hospital Universitario Puerta de Hierro and is a research scientist at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) and the Spanish cardiovascular research network (CIBERCV), led the study.
Dr. Pablo Garcia-Pava presented the findings of the first clinical trial using an amyloid-removing medication for the treatment of cardiac amyloidosis, coinciding with the publication of the study.
The study represents a significant step forward in the treatment of the Stiff-Heart Syndrome. Although existing treatments effectively prevent the generation of additional amyloid fibrils and delay disease progression, they do not directly eliminate any amyloid protein that has already accumulated in the heart.
Transthyretin-stabilizing therapy and strategies to control related cardiovascular problems are currently available as treatments. Heart transplantation is the only operation that can currently restore cardiac function in this illness.
Tafamidis, an oral transthyretin stabilizer, is the only medication licensed to treat transthyretin-related cardiac amyloidosis. Tafamidis improves survival and decreases hospitalizations; but, it does not alter illness symptoms that have already developed.
The preliminary findings of the research, which involved 40 patients from France, the Netherlands, Germany, and Spain and was overseen by Dr. Garca-Pava, reveal that the new medicine is safe and appears to reduce the quantity of amyloid protein accumulated in the heart.
The new drug, developed by the Swiss company Neurimmune, is an antibody that attaches to the transthyretin amyloid protein. The antibody was first isolated from memory B cells from healthy elderly people.
The antibody was utilized in the trial to stimulate the patients’ own defense mechanisms, which resulted in the removal of cardiac amyloid fibrils. Over a 12-month period, patients received the antibody intravenously in steadily increasing monthly doses.
“Patients who received higher antibody doses seemed to show a greater reduction in amyloid deposits in the heart and greater improvements in a range of cardiac parameters,” said Dr. García-Pavía.
The NEJM article concludes that the phase I proof-of-concept study demonstrates the safety of this treatment in patients and supports further clinical trials of this antibody.
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