Heart Reset

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves survival, in what researchers are calling “an unprecedented recovery of cardiac function.”

Currently, heart failure is irreversible without a transplant. But if this therapy shows similar results in future clinical trials, it could help heal the hearts of the one in four people who will eventually develop heart failure.

The treatment focuses on restoring a critical heart protein called cardiac bridging integrator 1 (cBIN1). “When cBIN1 is down, we know patients are not going to do well,” says Robin Shaw, director of the U’s Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) and co-senior author on the study. “It doesn’t take a rocket scientist to say, ‘What happens when we give it back?’ ”

The results were striking. Previous heart failure therapies have shown improvements of 5-10 percent. This therapy improved function by 30 percent. “It’s night and day,” Shaw adds.

“Even though the animals are still facing stress on the heart to induce heart failure, in animals that got the treatment, we saw recovery of heart function,” says TingTing Hong, associate professor of pharmacology and toxicology and CVRTI investigator at the U.

The team plans to apply for FDA approval for human clinical trials in fall 2025. “This human disease, which affects more than six million Americans—maybe this is something we can cure,” Hong says.