Navigation Links
Long-lasting gene therapy benefits advanced heart failure patients
Date:11/19/2013

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. 19 at the American Heart Association Scientific Sessions 2013.

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.


'/>"/>

Contact: Lauren Woods
lauren.woods@mountsinai.org
646-634-0869
The Mount Sinai Hospital / Mount Sinai School of Medicine
Source:Eurekalert  

Related medicine news :

1. Long-Lasting Gene Therapy Benefits Advanced Heart Failure Patients
2. Bumps in the road to developing long-lasting, single-injection nerve blocks
3. Five-Year Follow-Up Data on CERAMENT™
4. BONE VOID FILLER Demonstrates Long-Term Safety and Long-Lasting Bone Mineral Density Improvement in Osteoporotic Patients
5. Pain-free microneedle influenza vaccine is effective, long-lasting
6. Dr. Speron Announces the Release of Exparel for All Cosmetic Surgery for Long-lasting Pain Control
7. Parents Fighting May Have Long-Lasting Effect on Kids
8. Testosterone Therapy Safety a Top Priority at AAG Health
9. Depression in pregnancy: New study shows preferences for therapy over medication
10. A study led by CNIO validates a new anti-cancer therapy based on cell division
11. Orthobalance Physical Therapy Offers State-Of-The-Art Treatment for Balance Restoration and Fall Risk Management
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Long-lasting gene therapy benefits advanced heart failure patients
(Date:4/30/2016)... ... 2016 , ... Saturday, April 30, marks World Veterinary Day, ... health. The World Veterinary Association (WVA) and the World Organization for Animal Health ... with a One Health focus, which emphasizes how veterinarians pursue continuing education opportunities ...
(Date:4/29/2016)... ... April 30, 2016 , ... World ... the Pick Up Springboard, an automotive invention that improves the storage features of ... worth $162 billion," says Scott Cooper, CEO and Creative Director of World Patent ...
(Date:4/29/2016)... Sedona, Arizona (PRWEB) , ... April 29, 2016 ... ... Shamanic Healing and Spiritual Awakening , announces the addition Onnit brand Alpha BRAIN ... , The addition of Onnit brain and mood optimization products to the store ...
(Date:4/29/2016)... ... April 29, 2016 , ... The White ... repaying their loans, more information about their loan terms and accounts, and more ... loan debt, including federal and private loans, has reached $1.3 trillion, with 43 ...
(Date:4/29/2016)... ... April 29, 2016 , ... The ... the $30,000 Perlman Grand Prize of the 2016 Wharton Business Plan Competition ... Michelson People’s Choice Award, and the Committee Award for Most ‘Wow Factor,’ making ...
Breaking Medicine News(10 mins):
(Date:4/28/2016)... -- Research and Markets has announced the ... 2016-2020" report to their offering.      ... global plastic surgery products market is expected to grow ... , ,The growing adoption of laser in aesthetics is ... market. Lasers are used to treat a broad range ...
(Date:4/28/2016)... , April 28, 2016  The blood testing ... million dollars, according to Kalorama Information and The Freedonia ... immunoassays and nucleic acid testing.  The healthcare research firm ... progress in developing blood collection stations and in improving ... in Kalorama Information,s report, Blood Testing Market ...
(Date:4/28/2016)... Dr. Vivek Ahuja , George ... phen Schmidt Join the Growing Organization ... for life sciences, today announced key new leaders have joined the ... a growing business.  This will bolster the company,s safety business unit ... ArisGlobal in the position of Vice President - Safety. George has ...
Breaking Medicine Technology: