Navigation Links
New gene therapy proves effective in treating severe heart failure

Researchers at Mount Sinai School of Medicine have developed a new gene therapy that is safe and effective in reversing advanced heart failure. SERCA2a (produced as MYDICAR) is a gene therapy designed to stimulate production of an enzyme that enables the failing heart to pump more effectively. In a Phase II study, SERCA2a injection through a routine minimally invasive cardiac catheterization was safe and showed clinical benefit in treating this patient population and decreasing the severity of heart failure. The data were presented this week at the Heart Failure Congress of the European Society of Cardiology in Berlin.

"SERCA2a met the primary endpoints and appears to be safe and effective in people with advanced heart failure," said trial investigator Jill Kalman, MD, Associate Professor, Medicine, Cardiology, Director of the Cardiomyopathy Program, Mount Sinai School of Medicine. "There is a significant unmet need for treatments in this patient population, and these data indicate that SERCA2a is a promising option for them."

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 six months. The treatment is delivered directly to the patient's heart during a routine outpatient catheterization procedure.

Patients in the SERCA2a group demonstrated improvement or stabilization in symptoms, heart function, and severity of heart failure. They also saw an increase in time between cardiovascular events and a decrease in frequency of events. SERCA2a was found to be safe, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo.

SERCA2a was developed by a team led by Roger J. Hajjar, MD, Research Director of Mount Sinai's Wiener Family Cardiovascular Research Laboratories and the Arthur & Janet Ross Professor of Cardiology, Medicine, and Gene and Cell Medicine, Mount Sinai School of Medicine. The team discovered the landmark potential of the treatment in 1999 and has been pursuing its potential as a gene therapy target in state-of-the-art specially built pre-clinical laboratories at Mount Sinai.

"Mount Sinai Heart is committed to developing ground-breaking therapies and bringing them from bench to bedside," said Valentin Fuster, MD, Director of Mount Sinai Heart, the Zena and Michael A. Wiener Cardiovascular Institute and the Marie-Josee and Henry R. Kravis Center for Cardiovascular Health, The Mount Sinai Medical Center. "We look forward to further study of this important treatment."

According to the U.S. Centers for Disease Control & 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
The Mount Sinai Hospital / Mount Sinai School of Medicine

Related medicine news :

1. Career Training College, Pima Medical Institute, Offering Second Bachelor's Degree Program in Respiratory Therapy
2. Interruption of Menstrual Cycle by Chemotherapy Associated with Improved Survival in Early Breast Cancer
3. New Online Physical Therapy Marketing Academy Helps Physical Therapists Conquer The Web
4. Proton therapy carries precise, potent punch against childrens cancers
5. Dual Therapy Best for Preventing Bleeding After Heart Procedures
6. Blocking DNA repair protein could lead to targeted, safer cancer therapy
7. Targeted immunotherapy shows promise for metastatic breast, pancreatic cancers
8. AthletiCo Physical Therapy Enhances Work Rehabilitation Program by Offering Ergonomics and Job Analysis
9. Research finds surgery outperforms drug therapy in treatment of benign prostatic hyperplasia
10. Response to preoperative therapy may predict survival in pancreatic cancer patients
11. Making Your Way Through the Fog of Chemotherapy
Post Your Comments:
(Date:11/26/2015)... ... November 26, 2015 , ... Health-E-minds ... collaborated with Women’s Web – an online resource for Indian women looking ... and emotional well-being relationship, life balance, stress, professional development, and lifestyle. Health-E-minds ...
(Date:11/25/2015)... ... 25, 2015 , ... The holiday season is jam-packed with ... palates of attendees is of the utmost importance. Whether you are cooking at ... these recipes a try this holiday season. , Turkey Croquettes , ...
(Date:11/25/2015)... ... November 25, 2015 , ... Dr. John Pierce, Medical Director at the Ageless ... treatment with the Capillus272™ Pro laser therapy cap. FDA cleared for safety and efficacy, ... without the need for surgery, prescription pills, or topical foams. , “Capillus272™ Pro ...
(Date:11/25/2015)... ... 25, 2015 , ... According to an article published November 10th ... widely heralded as a breakthrough for performing hernia repairs. The article explains that the ... is that it can greatly reduce the pain that a patient might otherwise experience ...
(Date:11/25/2015)... ... November 25, 2015 , ... ... athletic programs, launches new Wimbledon Athletics Facebook page to educate the ... for unsuspected cardiac abnormalities. About 2,000 people under the age of 25 die ...
Breaking Medicine News(10 mins):
(Date:11/26/2015)... Nov. 26, 2015 Research and Markets ( ... "Advanced Wound Care Market by Type (Dressings, Therapy Devices, ... (In-Patient Facility, Out-Patient Facility), and Geography - Global Forecast ... --> --> The purpose ... and forecast of the global advanced wound care market. ...
(Date:11/26/2015)... India , November 26, 2015 ... --> adds ... Report" and "Investigation Report on China ... and 2021 forecasts data and information ... . ...
(Date:11/26/2015)... , Nov. 26, 2015 Research ... addition of the "2016 Future Horizons and ... (TDM) Market: Supplier Shares, Country Segment Forecasts, Competitive ... --> --> ... analysis of the Italian therapeutic drug monitoring market, ...
Breaking Medicine Technology: