Scientists have achieved what was once thought impossible: reversing heart failure and restoring cardiac function using a novel gene therapy. In a landmark study, researchers demonstrated dramatic improvements in heart function and survival rates in pigs with heart failure, offering hope for the millions who will develop this devastating condition.
Published in npj Regenerative Medicine | Estimated reading time: 5 minutes
The breakthrough centers on a critical heart protein called cardiac bridging integrator 1 (cBIN1), which researchers at the University of Utah Health restored through gene therapy. “When cBIN1 is down, we know patients are not going to do well,” explains Dr. Robin Shaw, director of the Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) and co-senior author of the study.
Currently, heart failure remains irreversible without a transplant. Most treatments focus on reducing cardiac stress and slowing disease progression. However, this new approach could fundamentally change how we treat the condition that will affect one in four people during their lifetime.
Unprecedented Recovery
The research team used a harmless virus to deliver an extra copy of the cBIN1 gene to heart cells through the bloodstream. The results were remarkable: all four treated pigs survived the entire six-month study period, despite having a condition that typically leads to death within months. More impressively, the therapy improved heart function by 30% – far exceeding the typical 5-10% improvements seen in previous treatments.
Dr. TingTing Hong, associate professor at the University and co-senior author, emphasizes the significance of the physical changes observed: “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 and that the heart also stabilizes or shrinks. We call this reverse remodeling. It’s going back to what the normal heart should look like.”
Key Terms
Gene Therapy
A technique that uses genes to treat or prevent disease by introducing genetic material into cells.
cBIN1 (Cardiac Bridging Integrator 1)
A critical protein that acts as a cellular scaffold in heart cells, organizing other proteins essential for proper heart function.
Reverse Remodeling
The process where damaged heart tissue returns toward its normal structure and function.
Test Your Knowledge
Currently, heart failure is irreversible without a heart transplant, with most treatments aimed at reducing stress on the heart and slowing disease progression. How much did the gene therapy improve heart function compared to previous treatments?
The therapy improved heart function by 30%, significantly higher than the typical 5-10% improvements seen in previous treatments. What role does cBIN1 play in heart cells?
cBIN1 serves as a centralized signaling hub and scaffold that organizes multiple proteins essential for heart cell function. What are the next steps for this research before it could help human patients?
The therapy must pass toxicology testing and other safety measures, and researchers plan to apply for FDA approval for human clinical trials in fall 2025.
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