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

June 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


Rank 4 /5 (1 vote)
Tags

Related Stories
Relevant PhysicsForums posts

More news stories

Study finds that anti-diabetic medication can prevent the long-term effects of maternal obesity

In a study to be presented today at the Society for Maternal-Fetal Medicine's annual meeting, The Pregnancy Meeting, in Dallas, Texas, researchers will report findings that show that short therapy with the anti-diabetic medication ...

Medicine & Health / Health

created 17 hours ago | popularity 5 / 5 (1) | comments 1

FDA-approved drug rapidly clears amyloid from the brain, reverses Alzheimer's symptoms in mice

Neuroscientists at Case Western Reserve University School of Medicine have made a dramatic breakthrough in their efforts to find a cure for Alzheimer's disease. The researchers' findings, published in the journal Science, show t ...

Medicine & Health / Neuroscience

created Feb 09, 2012 | popularity 4.9 / 5 (51) | comments 20 | with audio podcast

Steroid injections prove effective in treatment of lumbar disc herniations

The use of epidural steroid injections may be a more efficient treatment option for lumbar disc herniations, according to research presented today at the American Orthopaedic Society for Sports Medicine's Specialty Day in ...

Medicine & Health / Health

created 17 hours ago | popularity 5 / 5 (1) | comments 0

Amateur football players not always keen on returning to play after ACL injuries

Despite the known success rates of reconstructive Anterior Cruciate Ligament (ACL) surgery, the number of high school and collegiate football players returning to play may not be as high as anticipated, say researchers presenting ...

Medicine & Health / Health

created 17 hours ago | popularity not rated yet | comments 0

PRP treatment aids healing of elbow injuries say researchers

As elbow injuries continue to rise, especially in pitchers, procedures to help treat and get players back in the game quickly have been difficult to come by. However, a newer treatment called platelet rich plasma (PRP) may ...

Medicine & Health / Other

created 17 hours ago | popularity 5 / 5 (1) | comments 0


Walney offshore wind farm is world's biggest (for now)

(PhysOrg.com) -- The Walney wind farm on the Irish Sea--characterized by high tides, waves and windy weather--officially opened this week. The farm is treated in the press as a very big deal as the Walney ...

GPS court ruling leaves US phone tracking unclear

A US Supreme Court decision requiring a warrant to place a GPS device on the car of a criminal suspect leaves unresolved the bigger issue of police tracking using mobile phones, legal experts say.

Europeans protest controversial Internet pact

Tens of thousands of people marched in protests in more than a dozen European cities Saturday against a controversial anti-online piracy pact that critics say could curtail Internet freedom.

Europe stakes billion-dollar bet on new rocket

A pencil-slim rocket is scheduled to lift into space from South America on Monday, carrying a billion-dollar bet that Europe can grab a juicy slice of the market to place satellites in low orbit.

Netflix settlement trims 14 pct off 4Q earnings

(AP) -- Netflix pressed the rewind button on its fourth-quarter earnings after settling allegations that the video subscription service violated a consumer-privacy law.

Navy to begin tests on electromagnetic railgun prototype launcher

The Office of Naval Research (ONR)'s Electromagnetic (EM) Railgun program will take an important step forward in the coming weeks when the first industry railgun prototype launcher is tested at a facility ...