Washington: American researches have unveiled a new gene therapy that effectively treats severe heart failure. The SERCA2a (produced as MYDICAR), developed by researchers at Mount Sinai School of Medicine, is designed to stimulate production of an enzyme that enables the failing heart to pump more effectively.
"There is a significant unmet need for treatments in this patient population, and these data indicate that SERCA2a is a promising option for them." The CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial is a randomized, double-blind, placebo-controlled study, which enrolled 39 patients with advanced heart failure to study the safety and efficacy of SERCA2a. Patients were randomized to receive SERCA2a gene delivery in one of three doses or placebo and were evaluated over six months.
The treatment is delivered directly to the patient’s heart during a routine outpatient catheterization procedure. Patients in the SERCA2a group demonstrated improvement or stabilization in symptoms, heart function, and severity of heart failure. They also saw an increase in time between cardiovascular events and a decrease in frequency of events. SERCA2a was found to be safe, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo. ANI
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