Medicine's Next Big Thing: Gene Therapy for Failing Hearts?

February 26, 2010 12:00:00 AM PST
Seventeen Americans die every day waiting for a transplant that could save their lives. Now, gene therapy is being tested to give hope to those waiting on a heart. The cutting edge of medicine may get failing hearts beating normally again.Four-thousand people are waiting for a heart, and 450 people died last year on the transplant list.

Robert Beaubien doesn't want to be the next victim. He was born with congenital heart disease. Now 34, he sleeps, works, eats and exercises at the hospital.

"I'm in a 20x20 room with really bad air," Beaubien told Ivanhoe. "It gets too cold. It gets too hot. It's crazy. There's just things that will drive you nuts. If I don't stay mentally strong, I won't make that next trip. That's just the way it is."

He's been in and out of medical facilities for 34 years. Once a personal trainer, he's now a full-time patient. "All my organs failed," Beaubien said. "Following that, liver, lung, kidney, everything."

Molecular physiologist Todd Herron hopes that gene therapy will buy patients like Beaubien more time.

"What we're hoping to do is to eventually deliver genes to a failing heart," Dr. Herron, an assistant research professor in the Department of Molecular & Integrative Physiology at the University of Michigan in Ann Arbor, told Ivanhoe.

Dr. Herron is using the common cold virus to transport genes to heart muscle. The gene therapy turns on a protein that makes heart muscle cells contract more regularly. Dr. Herron says failing hearts could improve within 48 hours.

Beaubien was one of the lucky ones and received a heart. Doctors are hopeful gene therapy could help the thousands more who are still on the waiting list.

"Our hope is we could eliminate transplantation entirely," Eric Devaney, M.D., a pediatric cardiac surgeon at the University of Michigan in Ann Arbor, told Ivanhoe.

Gene therapy could help the 5 million people who suffer from heart failure, possibly saving $30 billion dollars each year in associated health care costs.


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