Navigation Links
Mount Sinai researchers develop new gene therapy for heart failure
Date:6/28/2011

Researchers at Mount Sinai School of Medicine have found in a Phase II trial that a gene therapy developed at Mount Sinai stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called SERCA2a, experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations, addressing a critical unmet need in this population. The data are published online in the June 27 issue of the American Heart Association journal Circulation.

SERCA2a is delivered via an adeno-associated virus vectoran inactive virus that acts as a medication transporterinto cardiac cells. The therapy stimulates production of an enzyme within these cells that enables the heart to pump more effectively in people with advanced heart failure. After one year, patients who were administered a high dose SERCA2a demonstrated improvement or stabilization. Gene therapy with SERCA2a was also found to be safe in this sick patient population, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo.

"Few treatment options have shown such improved clinical outcomes in this patient population in the last decade," said Roger J. Hajjar, MD, Research Director of Mount Sinai's Wiener Family Cardiovascular Research Laboratories, and the Arthur and Janet C. Ross Professor of Medicine, and Gene and Cell Medicine, Mount Sinai School of Medicine. "This study establishes a new paradigm for the treatment of heart failure by clinically validating SERCA2a as a novel target. In addition, by showing that adeno-associated vectors are safe to use in patients with advanced heart failure, this study ushers a new era for gene therapy for the treatment of failing hearts."

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 one year. The treatment is delivered directly to the patient's heart during a routine outpatient cardiac catheterization procedure.

Patients in the high-dose SERCA2a group demonstrated improvement and/or stabilization in symptoms, overall heart function, biomarker activity, and ventricular mechanics and function. They also saw a dramatic reduction in cardiovascular hospitalizations, averaging 0.4 days versus 4.5 days in the placebo group.

"Even though heart failure mortality has decreased over the last decade with the help of standard pharmacological and device therapies, patients with advanced heart failure continue to die at high rates. The CUPID trial offers a new therapeutic option for these patients," said Dr. Hajjar.

Led by Dr. Hajjar, the Mount Sinai team discovered the landmark potential of the cardiac-specific target in 1999 and has been pursuing its potential as a treatment delivered via gene therapy in state-of-the-art custom built laboratories at Mount Sinai School of Medicine in New York.

According to the U.S. Centers for Disease Control and Prevention, about 5.8 million Americans suffer from heart failure, and 670,000 new cases are diagnosed each year. One in five people who have heart failure die within one year of diagnosis. In 2010, heart failure will cost the United States $39.2 billion, including the cost of health care services, medications, and lost productivity. Heart failure is most often treated with aggressive medical and device therapy, but has no cure. The most common symptoms of heart failure are shortness of breath, feeling tired, and swelling in the ankles, feet, legs, and sometimes the abdomen.


'/>"/>

Contact: Mount Sinai Press Office
newsmedia@mssm.edu
212-241-9200
The Mount Sinai Hospital / Mount Sinai School of Medicine
Source:Eurekalert

Related biology news :

1. Large numbers of birth defects seen near mountaintop mining operations
2. Mountain pine beetle activity may impact snow accumulation and melt, says CU-Boulder study
3. Mountain pine beetle marching east from Alberta
4. Human virus linked to deaths of endangered mountain gorillas
5. UCLA researchers engineer E. coli to produce record-setting amounts of alternative fuel
6. Recipe for family mealtimes calls for 3 ingredients in the right amounts
7. Islands in the sky: How isolated are mountain top plant populations?
8. New method will triple amount of genetic information from newborn blood spot screenings
9. Winter sports threaten indigenous mountain birds
10. The amount of adipose tissue should be taken into account in the fight against obesity
11. New annotated database sifts through mountains of sequencing data to find gene promoters
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/5/2017)... LONDON , April 4, 2017 KEY ... is anticipated to expand at a CAGR of 25.76% ... neurodegenerative diseases is the primary factor for the growth ... full report: https://www.reportbuyer.com/product/4807905/ MARKET INSIGHTS The ... of product, technology, application, and geography. The stem cell ...
(Date:3/30/2017)... YORK , March 30, 2017 Trends, ... type (physiological and behavioral), by technology (fingerprint, AFIS, iris ... voice recognition, and others), by end use industry (government ... and immigration, financial and banking, and others), and by ... Europe , Asia Pacific , ...
(Date:3/24/2017)... Mar 24, 2017 Research and Markets has ... Market Analysis & Trends - Industry Forecast to 2025" report ... ... at a CAGR of around 15.1% over the next decade to ... report analyzes the market estimates and forecasts for all the given ...
Breaking Biology News(10 mins):
(Date:9/12/2017)... ... September 12, 2017 , ... ... announced the release of their latest product LabCODE™, a novel Lab Information ... LabCODE™ offers pre-built capabilities for Biobanking, Pharmacokinetics, Pharmacogenetics, Next Generation Sequencing, Flow ...
(Date:9/12/2017)... ... 2017 , ... VetStem Biopharma, Inc. , announced that a major new ... license to that patent. This patent covers methods for isolating stem ... stem cell with matrix materials, and incorporating the stem cells in an implant. ...
(Date:9/12/2017)... ... , ... An August 30th article on Daily Express explores a ... aging and overweight patients suffer from enlarged veins (genetics are also an important component), ... , The Washington State clinic Northwest Vein & Aesthetic Center notes that patients troubled ...
(Date:9/12/2017)... ... September 12, 2017 , ... OAI, ... semiconductor, MEMS, and microfluidics industries, announced today that multiple units of their advanced, ... design approach adds the flexibility required to meet the customers’ specific production process. ...
Breaking Biology Technology: