A groundbreaking gene therapy has demonstrated the potential to reverse heart failure and restore heart function in a large animal model. The treatment enhances the heart’s ability to pump blood and significantly improves survival rates, marking what researchers describe as “an unprecedented recovery of cardiac function.”
Currently, heart failure is considered irreversible. Most treatments aim to alleviate the stress on the heart and slow the progression of the disease, which is often fatal without a heart transplant. However, if this new gene therapy delivers similar results in human trials, it could offer hope for the millions of people at risk of developing heart failure in the future.
The focus of the research was on a key heart protein, cardiac bridging integrator 1 (cBIN1), which is found at lower levels in patients with heart failure. Lower cBIN1 levels have been linked to worsened disease outcomes. “When cBIN1 is down, we know patients are not going to do well,” said Dr. Robin Shaw, MD, PhD, director of the Nora Eccles Harrison Cardiovascular Research and Training Institute at the University of Utah and co-senior author of the study. “It doesn’t take a rocket scientist to say, ‘What happens when we give it back?’”
To address this, the team used a harmless virus commonly employed in gene therapy to deliver an extra copy of the cBIN1 gene to heart cells. They injected the virus into the bloodstream of pigs with heart failure, where it traveled to the heart and delivered the cBIN1 gene directly into the heart cells.
In this animal model, heart failure typically leads to death within a few months. However, all four pigs that received the gene therapy survived for at least six months—the endpoint of the study. The researchers believe that cBIN1’s ability to restore heart function is due to its role as a structural scaffold, interacting with proteins essential for heart muscle function.
“cBIN1 serves as a centralized signaling hub, which regulates multiple downstream proteins,” explained Dr. Jing Li, PhD, associate instructor at CVRTI and the study’s lead author. “By organizing the heart cell, cBIN1 helps restore critical functions.” The therapy seemed to improve heart function at the cellular level, with better-structured heart cells and proteins. The team hopes that cBIN1’s ability to regulate heart cell architecture could lay the foundation for a new approach to treating heart failure by targeting the muscle cells themselves.
The researchers, in collaboration with industry partner TikkunLev Therapeutics, are now adapting the therapy for human use. They plan to seek FDA approval for clinical trials in 2025. However, the therapy still needs to undergo toxicology testing and additional safety evaluations. Additionally, as with many gene therapies, there is a risk that some patients may have a natural immunity to the virus used in the treatment.
Despite these challenges, the researchers are optimistic. “When you see large animal data that closely mirrors human physiology, it makes you think,” said Dr. Hong, a co-author of the study. “Heart failure affects more than six million Americans—this might be the breakthrough that leads to a cure.”
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