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Gene therapy could aid transplant patients

January 26, 2010 12:00:00 AM PST
Seventeen Americans die every day waiting for a transplant that could save their lives. Doctors are using gene therapy and the cold virus to keep transplant patients healthier while they wait.Waiting on the list for an organ transplant can take its toll physically and psychologically.

But now, there's hope for patients with failing hearts. Cutting edge gene therapy may help get those 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 at 34, he sleeps, works, eats and exercises at the hospital.

"I'm in a 20 by 20 room with really bad air. It gets too cold. It gets too hot. It's just crazy. I mean, 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," Beaubien described.

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. Heart failure and following that, liver, lung, kidneys, everything," Beaubien said.

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

"What we're hoping to do is to eventually be able to deliver genes directly to a failing heart," explained Herron.

Herron is using the common cold virus to transport genes to the heart muscle. The gene therapy turns on a protein that makes heart muscle cells contract more regularly. 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 would be that we could eliminate transplantation entirely," said Herron.

Gene therapy could help the five million people who suffer from heart failure and possibly save $30 billion each year in healthcare costs.


TREATING HEART FAILURE WITH GENES?: A new study by scientists at the University of Michigan and University of Minnesota shows gene therapy may be used to improve an ailing heart's ability to contract properly. In addition to showing gene therapy's potential for reversing heart failure, it offers a new glimpse into a time where "closed heart surgery" using gene therapy is a possibility. "We hope that our study will lead some day to the development of new genetic-based therapies for heart failure patients," Todd J. Herron, Ph.D., assistant research professor in the Department of Molecular & Integrative Physiology at the University of Michigan in Ann Arbor, told Ivanhoe.

Dr. Herron and colleagues treated heart muscle cells from the failing hearts of rabbits and humans with a virus designed to carry a gene which produces a protein that enables heart cells to contract normally. Heart cells treated with the gene to express the fast molecular motor contracted better, while those treated with the gene to express the slow molecular motor were unaffected. The gene is injected into the heart through a catheter inserted into a blood vessel. Ideally, researchers say it would be a one-time treatment. "Our hope is we could eliminate heart transplants completely," Eric Devaney, M.D., a pediatric cardiac surgeon at the University of Michigan in Ann Arbor, told Ivanhoe.

BACKGROUND: According to the U.S. Centers for Disease Control and Prevention, heart failure is a condition where the heart cannot pump enough blood and oxygen to meet the needs of other organs. About 5 million people in the U.S. have heart failure, and about 55,000 new cases are diagnosed each year. More than 287,000 people die each year of heart failure. The most common causes of heart failure are coronary artery disease, hypertension or high blood pressure and diabetes. Current treatments usually involve medicines such as ACE inhibitors, diuretics, digoxin and beta blockers.