First gene therapy for heart failure offered at NewYork-Presbyterian/Columbia

Jun 18, 2008

[B]NYC's only medical center offering promising therapy as part of CUPID clinical trial[/B]
Could injecting a gene into a patient with severe heart failure reverse their disabling and life-threatening condition? Physician-scientists are setting out to answer that question in a first-ever clinical trial of gene therapy to treat severe heart failure.

NewYork-Presbyterian Hospital/Columbia University Medical Center is the only center in the New York City area where the therapy is currently available.

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

"This new therapy seeks to replenish the levels of this enzyme by introducing the gene for SERCA2a, which is depressed in these patients. If proven effective, this approach could be an alternative to heart transplant for patients without any other options," 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. She also is professor of medicine at Columbia University College of Physicians and Surgeons.

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, the most common vector 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 five million people in the U.S. have heart failure. Patients with severe form of the disease have trouble breathing because the heart cannot pump fluid out of their lungs. Seventy 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.

Source: New York- Presbyterian Hospital/Columbia University Medical Center

Explore further: Experts call for higher exam pass marks to close performance gap between international and UK medical graduates

add to favorites email to friend print save as pdf

Related Stories

Enzyme controls transport of genomic building blocks

Mar 06, 2014

Our DNA and its architecture are duplicated every time our cells divide. Histone proteins are key building blocks of this architecture and contain crucial information that regulates our genes. Danish researchers ...

One protein, two important roles

Jan 29, 2014

The molecular pathways involved in the cell cycle are complex and difficult to control. Better understanding of cell control mechanisms, and why they go wrong in cancer, could help open the door to more effective ...

Recommended for you

Obese British man in court fight for surgery

Jul 11, 2011

A British man weighing 22 stone (139 kilograms, 306 pounds) launched a court appeal Monday against a decision to refuse him state-funded obesity surgery because he is not fat enough.

2008 crisis spurred rise in suicides in Europe

Jul 08, 2011

The financial crisis that began to hit Europe in mid-2008 reversed a steady, years-long fall in suicides among people of working age, according to a letter published on Friday by The Lancet.

New food labels dished up to keep Europe healthy

Jul 06, 2011

A groundbreaking deal on compulsory new food labels Wednesday is set to give Europeans clear information on the nutritional and energy content of products, as well as country of origin.

Overweight men have poorer sperm count

Jul 04, 2011

Overweight or obese men, like their female counterparts, have a lower chance of becoming a parent, according to a comparison of sperm quality presented at a European fertility meeting Monday.

User comments : 0

More news stories

Treating depression in Parkinson's patients

A group of scientists from the University of Kentucky College of Medicine and the Sanders-Brown Center on Aging has found interesting new information in a study on depression and neuropsychological function in Parkinson's ...

Health care site flagged in Heartbleed review

People with accounts on the enrollment website for President Barack Obama's signature health care law are being told to change their passwords following an administration-wide review of the government's vulnerability to the ...

Airbnb rental site raises $450 mn

Online lodging listings website Airbnb inked a $450 million funding deal with investors led by TPG, a source close to the matter said Friday.