Transthyretin cardiac amyloidosis is a disease characterized by the deposit of amyloid protein in the heart, as a result of which the walls of this organ become thicker and more rigid, which is why it is also known as stiff heart syndrome. Those affected also experience fluid retention, arrhythmias and fatigue, and the accumulation of this substance in the heart causes heart failure, and can even cause death.
Stiff heart syndrome is associated with aging, although its origin may be genetic. Available treatments slow the progression of the disease and prevent further accumulation of amyloid protein, but they do not eliminate that which has already accumulated. This is the case of tafamidis, an oral drug that was approved in Europe in 2019 that prolongs the life of these patients –whose median survival without treatment is only 3 years– and reduces hospitalizations, but does not improve the disease. Now, the results of a new study with an antibody –NI006– developed by the Swiss laboratory Neurimmune that have been published in The New England Journal of Medicine (NEJM) can completely change the prognosis of these patients.
Dr. Pablo Garcia-Pavía, Head of Familial Cardiopathies at the Puerta de Hierro University Hospital, researcher at the National Center for Cardiovascular Research (CNIC) and CIBER Cardiovascular (CIBERCV) and one of the world’s leading experts on this disease has also presented the Results of this first clinical trial with an amyloid-removing drug against transthyretin cardiac amyloidosis at the Heart Failure Congress of the European Society of Cardiology held in Prague.
Stimulate the immune system to remove amyloid from the heart
NI006 is an antibody with the ability to bind to amyloid substance that has been identified from the analysis of type B memory cells from healthy very old people. The study in which it has been tested has been coordinated by Dr. García-Pavía at the Puerta de Hierro Hospital and 40 patients from France, the Netherlands, Germany and Spain have participated. Initial results from him show that the drug is safe and appears to reduce the amount of amyloid deposited in the heart.
“Patients who received higher doses of the drug appeared to have a greater reduction in amyloid deposits in the heart and greater improvement in various cardiac parameters.”
The researchers used this antibody to stimulate the immune system of the patients and thus achieve the elimination of cardiac amyloid. As explained in the article published in NEJM, the drug was administered intravenously at progressively higher doses on a monthly basis for 12 months. “Patients who received more doses of the drug apparently had a greater reduction in amyloid deposits in the heart and greater improvement in various cardiac parameters,” says Dr. García-Pavía.
The conclusions in the article indicate that this phase I trial demonstrates the safety profile of the new drug in these patients and supports further clinical research with the product so that it can be used in the treatment of people with stiff heart syndrome.
Dr. García-Pavía’s group at Hospital Puerta de Hierro is one of the most prestigious internationally in the diagnosis and treatment of cardiac amyloidosis and demonstrated years ago that this disease, which was previously considered very rare, is a of the most common causes of heart failure in people older than 65 years.
Source: National Center for Cardiovascular Research (CNIC)
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