New gene therapy to effectively treat severe heart failure

American researchers have unveiled a new gene therapy that effectively treats severe heart failure.

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.
In a Phase II study, SERCA2a injection through a routine minimally invasive cardiac catheterization was safe and showed clinical benefit in treating this patient population and decreasing the severity of heart failure.

The data were recently presented at the Heart Failure Congress of the European Society of Cardiology in Berlin.

Trial investigator Jill Kalman, Associate Professor, Medicine, Cardiology, Director of the Cardiomyopathy Program, Mount Sinai School of Medicine, said: "SERCA2a met the primary endpoints and appears to be safe and effective in people with advanced heart failure.
"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.


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