Imma Perfetto
Cosmos science journalist
A new gene therapy may give hope to the more than 64 million people living with heart failure worldwide.
Robin Shaw, director of the Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) at the University of Utah says: “In the history of heart failure research, we have not seen efficacy like this.”
Current treatment options for the disease are limited to reducing stress on the heart and slowing progression. Ultimately, the affected heart can be replaced through transplantation, but demand for organs far outstrips supply.
The new gene therapy, reported in a npj Regenerative Medicine paper, prevented heart failure (HF) from worsening and even improved some key measures of heart function in pig models.
Previous attempted therapies have resulted in 5-10% improvements to heart function. This gene therapy restores the function of the critical heart protein cardiac bridging integrator 1 (cBIN1) and improved function by 30%.
“When cBIN1 is down, we know patients are not going to do well,” says Shaw.
“It doesn’t take a rocket scientist to say, ‘What happens when we give it back?’”
A new copy of the cBIN1 gene was delivered to heart cells by packaging it up in a harmless adeno-associated virus. In the present study, it was injected once into the bloodstream of pigs.
This model of HF usually leads to death within just a few months, but all 4 pigs that received the gene therapy survived until the end of the 6-month study. Their hearts’ efficiency at pumping blood also increased to close to normal over that time.
“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 stabilises or shrinks,” says TingTing Hong, associate professor of pharmacology and toxicology at the University of Utah and co-senior author on the study.
“We call this reverse remodelling. It’s going back to what the normal heart should look like.
“When you see large animal data that’s really close to human physiology, it makes you think,” Hong says. “Maybe this is something we can cure.”
The team is currently adapting the gene therapy for use in humans and intend to apply for FDA approval for human clinical trial in 2025.
