Phase I Trial Demonstrates First Drug Therapy Can Improve Cardiac Function in Stiff Cardiac Syndrome

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human heart. Credit: copyright American Heart Association

Transthyretin-associated cardiac amyloidosis is a progressive disease characterized by deposition of amyloid protein fibrils in the heart. The deposition of amyloid fibrils causes the heart wall to thicken and stiffen, a condition also known as rigorous heart syndrome. Accumulation of amyloid fibrils leads to heart failure, and patients suffer from fluid retention, fatigue and arrhythmias. The disease can be caused by genetic mutations or associated with aging. The prognosis is poor, with an average survival time of only 3 years for untreated patients.

Well, a research result was published in a journal. New England Journal of Medicine (NEJM) promises to radically change the outlook for patients with this disease. The study was conducted by Pablo García-Pavia, director of the Department of Hereditary Heart Diseases at the Puerta de Hierro University Hospital and research fellow at the National Cardiovascular Research Center (CNIC) and the Spanish Cardiovascular Research Network (CIBERCV). Led by Dr. .

Concurrent with the publication of this study, Dr. Pablo García-Pavia today announced the results of the first clinical trial of an amyloid-removing drug for the treatment of cardiac amyloidosis.

This study represents a major advance in the treatment of this disease. Currently available treatments effectively prevent further amyloid fibril accumulation and slow disease progression, but do not directly remove amyloid protein already deposited in the heart.

Current treatment options include transthyretin-stabilizing therapy and measures to control associated cardiovascular complications. Currently, heart transplantation is the only intervention that can restore cardiac function in this disease.

The only drug approved for the treatment of transthyretin-associated cardiac amyloidosis is tafamidis, an oral transthyretin stabilizer. Tafamidis increases survival and reduces hospitalizations. However, it cannot reverse the symptoms of an already established disease.

Initial results from the trial, which included 40 patients in France, the Netherlands, Germany and Spain and was coordinated by Dr. García-Pavia, indicate that the new drug is safe and appears to reduce the amount of amyloid protein deposited in the body. is shown. heart.

The new drug, developed by Nulimune in Switzerland, is an antibody that binds to the transthyretin amyloid protein. This antibody was originally isolated from memory B cells obtained from healthy elderly individuals.

In this study, antibodies were used to stimulate the patient’s own defense system, resulting in clearance of cardiac amyloid fibrils. Antibodies were administered intravenously to patients in gradually increasing monthly doses for 12 months.

“Patients who received higher doses of antibody appeared to show greater reductions in amyloid deposits in the heart and greater improvements in a range of cardiac parameters,” said Dr. García-Pavia.

of NEJM The paper concludes that a phase I proof-of-concept study demonstrates the safety of this treatment in patients and supports further clinical trials of this antibody.

For more information:
Pablo Garcia-Pavía et al., Phase I study of antibody NI006 that depletes cardiac amyloid transthyretin, New England Journal of Medicine (2023). Doi:

Magazine information:
New England Journal of Medicine

Courtesy of Carlos III Cardiovascular Research Center (FSP)

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