Navigation Links
Long-Lasting Gene Therapy Benefits Advanced Heart Failure Patients
Date:11/19/2013

New York, New York (PRWEB) 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. 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.

Dr. Hajjar is the scientific cofounder of the company Celladon, which is developing AAV1/SERCA2a gene therapy for the treatment of heart failure. He holds equity in Celladon and receives financial compensation as a member of its advisory board.

About the Mount Sinai Health System
The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven member hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services—from community-based facilities to tertiary and quaternary care.

The System includes approximately 6,600 primary and specialty care physicians, 12-minority-owned free-standing ambulatory surgery centers, over 45 ambulatory practices throughout the five boroughs of New York City, Westchester, and Long Island, as well as 31 affiliated community health centers. Physicians are affiliated with the Icahn School of Medicine at Mount Sinai, which is ranked among the top 20 medical schools both in National Institutes of Health funding and by U.S. News & World Report.
For more information, visit http://www.mountsinai.org.
Find Mount Sinai on:
Facebook: http://www.facebook.com/mountsinainyc
Twitter @mountsinainyc
YouTube: http://www.youtube.com/mountsinainy

Read the full story at http://www.prweb.com/releases/2013/11/prweb11341052.htm.


'/>"/>
Source: PRWeb
Copyright©2012 Vocus, Inc.
All rights reserved

Related medicine news :

1. Bumps in the road to developing long-lasting, single-injection nerve blocks
2. Five-Year Follow-Up Data on CERAMENT™
3. BONE VOID FILLER Demonstrates Long-Term Safety and Long-Lasting Bone Mineral Density Improvement in Osteoporotic Patients
4. Pain-free microneedle influenza vaccine is effective, long-lasting
5. Dr. Speron Announces the Release of Exparel for All Cosmetic Surgery for Long-lasting Pain Control
6. Parents Fighting May Have Long-Lasting Effect on Kids
7. Testosterone Therapy Safety a Top Priority at AAG Health
8. Depression in pregnancy: New study shows preferences for therapy over medication
9. A study led by CNIO validates a new anti-cancer therapy based on cell division
10. Orthobalance Physical Therapy Offers State-Of-The-Art Treatment for Balance Restoration and Fall Risk Management
11. Italian study examines clinical predictors of acute urinary symptoms after radiotherapy for prostate
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/25/2016)... , ... June 25, 2016 , ... "With 30 hand-drawn ... specific project," said Christina Austin - CEO of Pixel Film Studios. , ProHand ... all within Final Cut Pro X . Simply select a ProHand generator and ...
(Date:6/25/2016)... Montreal, Canada (PRWEB) , ... June 25, 2016 , ... ... the pursuit of success. In terms of the latter, setting the bar too high ... low, risk more than just slow progress toward their goal. , Research from ...
(Date:6/24/2016)... Texas (PRWEB) , ... June 24, 2016 , ... ... International Conference and Scientific Sessions in Dallas that it will receive two significant ... of the grants came as PHA marked its 25th anniversary by recognizing patients, ...
(Date:6/24/2016)... , ... June 24, 2016 , ... People across the ... Genome magazine’s Code Talker Award, an essay contest in which patients and their families ... to be presented at the 2016 National Society of Genetic Counselors (NSGC) Annual Education ...
(Date:6/24/2016)... ... ... National recruitment firm Slone Partners is pleased to announce the ... as Vice President of North American Capital Sales at HTG Molecular . ... team in the commercialization of the HTG EdgeSeq system and associated reagents in North ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... 24, 2016   Bay Area Lyme Foundation ... Dean Center for Tick Borne Illness , Harvard ... MIT Hacking Medicine, University of California, Berkeley, and ... the five finalists of Lyme Innovation , ... than 100 scientists, clinicians, researchers, entrepreneurs, and investors ...
(Date:6/24/2016)... SAN CLEMENTE, Calif. , June 24, 2016  American Respiratory ... testing company, is now able to perform sophisticated lung assessments in ... Medical Technologies , Inc. Patients are no longer ... to ndd,s EasyOne PRO ® , ARL patients like Jeanne R. ... testing done in the comfort of her own home. ...
(Date:6/24/2016)... , June 24, 2016  Arkis BioSciences, ... less invasive and more durable cerebrospinal fluid treatments, ... funding.  The Series-A funding is led by Innova ... Fund, and other private investors.  Arkis, new financing ... instrumentation and the market release of its in-licensed ...
Breaking Medicine Technology: