Gene therapy improved left ventricular and atrial function in heart failure by up to 25 percent

September 25, 2017, The Mount Sinai Hospital
Credit: CC0 Public Domain

Heart function improved by up to 25 percent in a trial using gene therapy to reverse cardiac damage from congestive heart failure in a large animal model, Mount Sinai researchers report. This is the first study using a novel vector for gene therapy to improve heart function in non-ischemic heart failure.

The results of the study will be published online in the Journal of the American College of Cardiology.

In , a weakened or damaged heart no longer pumps blood effectively. This potentially fatal disease affects almost 6 million Americans, according to the American Heart Association, and is a major cause of morbidity and mortality, especially in elderly patients. Despite this toll, there has been little progress toward any kind of cure. Novel therapeutic approaches, such as and cell therapy, hold the promise of complementing or replacing existing therapies for .

"Mount Sinai has performed pioneering work on gene therapy over the last decade, and this study shows that gene therapy is now a viable option for treating congestive heart failure," said the study's senior author, Roger Hajjar, MD, Director of the Cardiovascular Research Center and the Arthur and Janet C. Ross Professor of Medicine at the Icahn School of Medicine at Mount Sinai. "There is a critical need to explore new therapeutic avenues and approaches."

This study featured two independent experiments. The first established the safety of administering a therapeutic gene delivery vector, BNP116, created from an inactivated virus over three months, into 48 pigs without heart failure through the coronary arteries via catheterization using echocardiography. The second experiment examined the efficacy of the treatment in 13 pigs with severe heart failure induced by mitral regurgitation. Six pigs received the gene and 7 received a saline solution.

The researchers determined that the gene therapy was safe and significantly reversed heart failure by 25 percent in the left ventricle and by 20 percent in the left atrium. Heart failure often results in enlarged hearts, and the team found a 10 percent reduction of size in the affected animals. Heart failure in the cohort of pigs treated with saline worsened.

The research team plans to study the same gene therapy in a human trial starting next year. The gene vector has been approved by the Food and Drug Administration for clinical treatment.

Explore further: Safety of gene transfer to treat heart failure supports further clinical development

Related Stories

Safety of gene transfer to treat heart failure supports further clinical development

June 6, 2017
Based on the encouraging safety data that has emerged from multiple clinical trials that used different gene transfer approaches to improve heart function in patients suffering from heart failure with reduced ejection fraction, ...

Novel gene therapy works to reverse heart failure

November 13, 2013
Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested a powerful gene therapy, delivered directly into the heart, to reverse heart failure in large animal models.

Long-lasting gene therapy benefits advanced heart failure patients

November 19, 2013
Researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai reported the long-term benefits of a single dose of their gene therapy AAV1/SERCA2a in advanced heart failure patients on Nov. ...

Breathing in a new gene therapy to treat pulmonary hypertension

July 12, 2017
Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is ...

Gene transfer shows promise for treating heart failure

March 30, 2016
Use of intracoronary gene transfer among heart failure patients resulted in increased left ventricular function beyond standard heart failure therapy, according to a study published online by JAMA Cardiology.

Inhalable gene therapy may help pulmonary arterial hypertension patients

July 30, 2013
The deadly condition known as pulmonary arterial hypertension (PAH), which afflicts up to 150,000 Americans each year, may be reversible by using an inhalable gene therapy, report an international team of researchers led ...

Recommended for you

Drinking alcohol makes your heart race

March 18, 2018
The more alcohol you drink, the higher your heart rate gets, according to research presented today at EHRA 2018 Congress, organized by the European Society of Cardiology.

Study of nearly 300,000 people challenges the 'obesity paradox'

March 15, 2018
The idea that it might be possible to be overweight or obese but not at increased risk of heart disease, otherwise known as the "obesity paradox", has been challenged by a study of nearly 300,000 people published in in the ...

Mending broken hearts with cardiomyocyte molds

March 13, 2018
2.5 billion. That's approximately the number of times the human heart beats in 70 years. And sometimes during the course of its unrelenting contractions and relaxations, the heart muscle can no longer bear the strain.

Common infections a bigger heart disease and stroke risk than obesity

March 13, 2018
A major study into the impact of common infections leading to hospitalisation has found they may substantially increase the risk of heart attacks, strokes and in the longer term, death.

Aspirin prevents venous thromboembolism following major orthopedic surgeries, study finds

March 13, 2018
A multicentre, double-blind, randomized, controlled clinical trial of patients who underwent total hip or knee replacement surgery showed that aspirin was as effective as rivaroxaban, the standard anti-coagulation medication, ...

Barbershop-based healthcare study successfully lowers high blood pressure in African-American men

March 12, 2018
African-American men successfully lowered their high blood pressure to healthy levels when aided by a pharmacist and their local barber, according to a new study from the Smidt Heart Institute.


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.