News Column

Researchers' Work from Cardiovascular Research Center Focuses on Gene Therapy

June 26, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Biotechnology have been published. According to news originating from New York City, New York, by NewsRx correspondents, research stated, "The Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In Cardiac Disease (CUPID 1) study was a phase 1/phase 2 first-in-human clinical gene therapy trial using an adeno-associated virus serotype 1 (AAV1) vector carrying the sarcoplasmic reticulum calcium ATPase gene (AAV1/SERCA2a) in patients with advanced heart failure. The study explored potential benefits of the therapy at 12 months, and results were previously reported."

Our news journalists obtained a quote from the research from Cardiovascular Research Center, "To report long-term (3-year) clinical effects and transgene expression in the patients in CUPID 1. A total of 39 patients with advanced heart failure who were on stable, optimal heart failure therapy were randomized to receive intracoronary infusion of AAV1/SERCA2a in 1 of 3 doses (low-dose, 6x10(11) DNase-resistant particles; mid-dose, 3x10(12) DNase-resistant particles; and high-dose, 1x10(13) DNase-resistant particles) versus placebo. The following recurrent cardiovascular and terminal events were tracked for 3 years in all groups: myocardial infarction, worsening heart failure, heart failure-related hospitalization, ventricular assist device placement, cardiac transplantation, and death. The number of cardiovascular events, including death, was highest in the placebo group, high but delayed in the low- and mid-dose groups, and lowest in the high-dose group. Evidence of long-term transgene presence was also observed in high-dose patients. The risk of prespecified recurrent cardiovascular events was reduced by 82% in the high-dose versus placebo group (P=0.048). No safety concerns were noted during the 3-year follow-up."

According to the news editors, the research concluded: "After a single intracoronary infusion of AAV1/SERCA2a in patients with advanced heart failure, positive signals of cardiovascular events persist for years."

For more information on this research see: Long-Term Effects of AAV1/SERCA2a Gene Transfer in Patients With Severe Heart Failure Analysis of Recurrent Cardiovascular Events and Mortality. Circulation Research, 2014;114(1):101-108. Circulation Research can be contacted at: Lippincott Williams & Wilkins, 530 Walnut St, Philadelphia, PA 19106-3621, USA (see also Biotechnology).

The news correspondents report that additional information may be obtained from K. Zsebo, Icahn Sch Med Mt Sinai, Cardiovasc Res Center, New York, NY 10029, United States. Additional authors for this research include A. Yaroshinsky, J.J. Rudy, K. Wagner, B. Greenberg, M. Jessup and R.J. Hajjar.

Keywords for this news article include: Biotechnology, Cardiology, Gene Therapy, New York City, United States, Heart Disease, Heart Failure, Bioengineering, Cardiovascular Diseases, North and Central America

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Source: Gene Therapy Weekly

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