Korede Abdullah in Lagos
A landmark gene therapy has shown unprecedented results in reversing heart failure and restoring cardiac function in a large animal model.
The therapy, which increases cardiac bridging integrator 1 (cBIN1) levels, significantly improved heart function and survival rates.
Researchers focused on restoring cBIN1, a critical heart protein, which is lower in heart failure patients. “When cBIN1 is down, we know patients are not going to do well,”, says Robin Shaw, MD, PhD, director of the Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) at the University of Utah and a co-senior author on the study.
The team used a harmless virus to deliver the cBIN1 gene to heart cells in pigs with heart failure.
The results were remarkable: all four treated pigs survived for six months, with improved heart function and reduced dilation.
“In the history of heart failure research, we have not seen efficacy like this,” Shaw notes. Previous therapies showed 5-10% improvements, while cBIN1 gene therapy improved function by 30%.
The treated hearts’ efficiency at pumping blood increased over time, and the hearts remained less dilated.
“We saw recovery of heart function and reverse remodeling,” says Dr. TingTing Hong, co-senior author. The researchers believe cBIN1’s role as a scaffold interacting with heart muscle proteins is key to its success.
The team hopes to adapt the gene therapy for human use and apply for FDA approval in 2025. While challenges remain, the results are promising.
“Maybe this is something we can cure,” Hong says, referring to heart failure, which affects over six million Americans.
The study’s findings offer new hope for heart failure treatment, potentially introducing a paradigm shift in targeting heart muscle itself.
