Featured Research

from universities, journals, and other organizations

First Trial Of Gene Therapy For Advanced Heart Failure Shows Promising Results

Date:
November 11, 2008
Source:
New York- Presbyterian Hospital/Columbia University Medical Center
Summary:
Phase I results of the first clinical trial of gene therapy for patients with advanced heart failure show the approach to be promising, with improvements in several measures of the condition's severity.

Phase I results of the first clinical trial of gene therapy for patients with advanced heart failure show the approach to be promising, with improvements in several measures of the condition's severity.

In Phase I clinical trials, researchers test a new treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

Patients enrolled in the multicenter CUPID trial (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease) undergo a minimally invasive cardiac catheterization procedure which introduces a specially engineered gene that stimulates production of an enzyme necessary for the heart to pump more efficiently.

NewYork-Presbyterian Hospital/Columbia University Medical Center was the first to offer the therapy in the New York City area. The Hospital is now recruiting patients for the Phase II CUPID trial to further assess safety and effectiveness in patients with advanced heart failure.

Data from the Phase I trial, which was initiated in May of 2007, were presented at the American Heart Association (AHA) Scientific Sessions 2008 in New Orleans yesterday. Seven of nine patients who were given the drug showed improvements over six months in several areas: symptomatic (five patients), functional (four patients), biomarker (two patients) and left ventricular function/remodeling (six patients). Two patients with pre-existing antibodies to the viral vector delivery system did not show improvements. Importantly, the approach was shown to have an acceptable safety profile, as determined by an independent safety committee and by the study investigators.

"We are encouraged by these initial findings, which indicate that this therapy has the potential to help patients with advanced heart failure," says Dr. Donna Mancini, the study's principal investigator at NewYork-Presbyterian Hospital/Columbia University Medical Center, where she is medical director of cardiac transplantation and is professor of medicine at Columbia University College of Physicians and Surgeons.

The Phase II randomized, double-blind, placebo-controlled clinical trial will compare the therapy at two- or three-dose levels with placebo. CUPID is expected to enroll 46 patients with advanced heart failure at 13 U.S. hospitals.

Gene therapy is a technique for correcting defective genes responsible for disease development by inserting genes into a patient's cells and tissues. In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal" disease-causing gene. A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, one of the most common vectors is a non-pathogenic virus most people have been exposed to in adolescence that has been genetically altered to carry normal human DNA.

More than 5 million people in the U.S. have heart failure. Patients with severe form of the disease have trouble breathing because the heart muscle is not strong enough to pump fluid out of their lungs. Approximately 70 percent die of the disease within 10 years, and the five-year survival rate is less than 50 percent. Heart failure is the only cardiovascular disease whose incidence has been increasing rather than decreasing in recent years.

The multicenter national trial is funded and administered by the Celladon Corporation of La Jolla, Calif. The company has reported that the therapy, called MYDICAR®, has been shown to lead to significant improvements in heart function without significant safety concerns in numerous large-animal models of heart failure.

 


Story Source:

The above story is based on materials provided by New York- Presbyterian Hospital/Columbia University Medical Center. Note: Materials may be edited for content and length.


Cite This Page:

New York- Presbyterian Hospital/Columbia University Medical Center. "First Trial Of Gene Therapy For Advanced Heart Failure Shows Promising Results." ScienceDaily. ScienceDaily, 11 November 2008. <www.sciencedaily.com/releases/2008/11/081110171342.htm>.
New York- Presbyterian Hospital/Columbia University Medical Center. (2008, November 11). First Trial Of Gene Therapy For Advanced Heart Failure Shows Promising Results. ScienceDaily. Retrieved April 21, 2014 from www.sciencedaily.com/releases/2008/11/081110171342.htm
New York- Presbyterian Hospital/Columbia University Medical Center. "First Trial Of Gene Therapy For Advanced Heart Failure Shows Promising Results." ScienceDaily. www.sciencedaily.com/releases/2008/11/081110171342.htm (accessed April 21, 2014).

Share This



More Health & Medicine News

Monday, April 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Breakfast Foods Are Getting Pricier

Breakfast Foods Are Getting Pricier

AP (Apr. 21, 2014) — Breakfast is now being served with a side of sticker shock. The cost of morning staples like bacon, coffee and orange juice is on the rise because of global supply problems. (April 21) Video provided by AP
Powered by NewsLook.com
Nine-Month-Old Baby Can't Open His Mouth

Nine-Month-Old Baby Can't Open His Mouth

Newsy (Apr. 19, 2014) — Nine-month-old Wyatt Scott was born with a rare disorder called congenital trismus, which prevents him from opening his mouth. Video provided by Newsy
Powered by NewsLook.com
'Holy Grail' Of Weight Loss? New Find Could Be It

'Holy Grail' Of Weight Loss? New Find Could Be It

Newsy (Apr. 18, 2014) — In a potential breakthrough for future obesity treatments, scientists have used MRI scans to pinpoint brown fat in a living adult for the first time. Video provided by Newsy
Powered by NewsLook.com
Little Progress Made In Fighting Food Poisoning, CDC Says

Little Progress Made In Fighting Food Poisoning, CDC Says

Newsy (Apr. 18, 2014) — A new report shows rates of two foodborne infections increased in the U.S. in recent years, while salmonella actually dropped 9 percent. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins