Amyloid-removing drug hopes to treat stiff heart syndrome

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 fundamentally change the outlook for patients with this disease. The study was led by Dr. Pablo García Pavia, director of the Department of Hereditary Heart Diseases at the National Hospital. Puerta de Hierro University Hospital is a research scientist in National Cardiovascular Research Center (CNIC) and within the Spanish Cardiovascular Research Network (CIBERCV). 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 receiving higher doses of antibody appeared to show greater reductions in intracardiac amyloid deposition and greater improvements in a range of cardiac parameters. ”

Dr. Pablo Garcia-Pavia

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

Dr. Garcia Pavia is the world leader in transthyretin-associated cardiac amyloidosis and the leader of the European College of Cardiology guidelines for the diagnosis and treatment of this disease, which are being followed worldwide.

his group Puerta de Hierro Hospital An international reference in the field, it was discovered several years ago that this disease, previously thought to be very rare, is one of the most frequent causes of heart failure in people over the age of 65. Proven.


Carlos III Cardiovascular Research Center (FSP)

Reference magazine:

Garcia Pavia, P. other. (2023) Phase I study of antibody NI006 that depletes cardiac amyloid transthyretin. New England Journal of Medicine. Moa2303765.

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