Featured Research

from universities, journals, and other organizations

Long-lasting gene therapy benefits advanced heart failure patients

Date:
November 19, 2013
Source:
Mount Sinai Medical Center
Summary:
Researchers have reported the long-term benefits of a single dose of their gene therapy AAV1/SERCA2a in advanced heart failure patients.

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. 19 at the American Heart Association Scientific Sessions 2013.

Related Articles


The new long-term follow-up results from their initial Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In Cardiac Disease (CUPID 1) clinical trial found a one-time, high-dose injection of the AAV1/SERCA2a gene therapy results in the presence of the delivered SERCA2a gene up to 31 months in the cardiac tissue of heart failure patients.

In addition, study results show clinical event rates in gene therapy patients are significantly lower three years later compared to those patients receiving placebo. Also, patients experienced no negative side effects following gene therapy delivery at three-year follow-up.

"This study shows AAV1/SERCA2a gene therapy has long-lasting and beneficial effects for congestive heart failure patients allowing us to block the downward spiral of patients with severe heart failure, " says principal investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center and the Arthur & Janet C. Ross Professor of Medicine at Icahn School of Medicine at Mount Sinai, who developed the gene therapy approach.

The gene therapy uses a modified adeno-associated viral-vector derived from a parvovirus. The one-time gene therapy is injected through the coronary arteries of heart failure patients using catheters. It works by introducing healthy SERCA2a genes into cells. The delivery of the SERCA2a gene produces SERCA2a enzymes, which helps heart cells restore their proper use of calcium.

SERCA2a is an enzyme critical for proper pumping of calcium in calcium compartments within cells. SERCA2a dysfunction or reduced expression occurs in patients with heart failure. When SERCA2a is down-regulated, calcium stays longer in the cells than it should, and it induces pathways that lead to overgrowth of new and enlarged cells. This contributes to an enlarged heart in heart failure patients.

Previously, CUPID 1 study results showed the gene therapy to be clinically safe and effective for over 12 months with improved heart function status and left ventricular function, along with a significant decrease in recurrent cardiovascular events. CUPID 1 was the first-in human clinical gene therapy randomized, double-blind study which enrolled 39 patients with advanced heart failure.

"AAV1/SERCA2a gene therapy has been proven to be a safe and effective therapeutic intervention for advanced heart failure," says Dr. Hajjar. "Our long-term results support the potential use of AAV1/SERCA2a gene therapy as a new important additional tool for treating and managing advanced heart failure patients."

This study was presented as an Oral Session (Abstract 10667): Long Term Follow-up of Patients with Advanced Heart Failure Following a Single Intracoronary Infusion of AAV1/SERCA2a. In addition, on Nov. 19 Dr. Hajjar also presented at the AHA Scientific Sessions 2013 a Plenary talk entitled, "How the Postgenome Era Will Change the Practice of Cardiology" and discussed his work on targeted gene therapy for human heart failure.

In his Plenary talk, Dr Hajjar presented his new findings just published in the journal Science Translational Medicine on Nov. 13 that show delivery of small ubiquitin-related modifier 1 (SUMO-1), an important regulator of SERCA2a, in preclinical heart failure models improves cardiac contractility and prevents left ventricular dilatation -- two major aspects of heart failure. According to Dr. Hajjar, the transition of this SUMO-1 gene therapy from pigs to humans seems likely in the short-term. Also, Dr. Hajjar revealed that development of novel cardiotropic vectors may render cardiovascular gene therapy easier and less-invasive in the near future.


Story Source:

The above story is based on materials provided by Mount Sinai Medical Center. Note: Materials may be edited for content and length.


Cite This Page:

Mount Sinai Medical Center. "Long-lasting gene therapy benefits advanced heart failure patients." ScienceDaily. ScienceDaily, 19 November 2013. <www.sciencedaily.com/releases/2013/11/131119100927.htm>.
Mount Sinai Medical Center. (2013, November 19). Long-lasting gene therapy benefits advanced heart failure patients. ScienceDaily. Retrieved November 23, 2014 from www.sciencedaily.com/releases/2013/11/131119100927.htm
Mount Sinai Medical Center. "Long-lasting gene therapy benefits advanced heart failure patients." ScienceDaily. www.sciencedaily.com/releases/2013/11/131119100927.htm (accessed November 23, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, November 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

WFP: Ebola Risks Heightened Among Women Throughout Africa

WFP: Ebola Risks Heightened Among Women Throughout Africa

AFP (Nov. 21, 2014) Having children has always been a frightening prospect in Sierra Leone, the world's most dangerous place to give birth, but Ebola has presented an alarming new threat for expectant mothers. Duration: 00:37 Video provided by AFP
Powered by NewsLook.com
Could Your Genes Be The Reason You're Single?

Could Your Genes Be The Reason You're Single?

Newsy (Nov. 21, 2014) Researchers in Beijing discovered a gene called 5-HTA1, and carriers are reportedly 20 percent more likely to be single. Video provided by Newsy
Powered by NewsLook.com
Raw: Paralyzed Marine Walks With Robotic Braces

Raw: Paralyzed Marine Walks With Robotic Braces

AP (Nov. 21, 2014) Marine Corps officials say a special operations officer left paralyzed by a sniper's bullet in Afghanistan walked using robotic leg braces in a ceremony to award him a Bronze Star. (Nov. 21) Video provided by AP
Powered by NewsLook.com
Milestone Birthdays Can Bring Existential Crisis, Study Says

Milestone Birthdays Can Bring Existential Crisis, Study Says

Newsy (Nov. 21, 2014) Researchers find that as people approach new decades in their lives they make bigger life decisions. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins