New gene therapy leads to unprecedented recovery from heart failure in animal study.

A new study suggests that heart failure, which has historically been irreversible, could someday change.

In a large animal study, scientists at the University of Utah successfully reversed the effects of heart failure using a new gene therapy.

A critical heart protein, cBIN1, was found to have low levels in pigs with heart failure, according to a study.

According to a university press release, the scientists injected a harmless virus into the pigs' bloodstreams to deliver the cBIN1 gene into their heart cells.

The pigs lived for six months during the study, despite being predicted to die from heart failure without gene therapy.

The IV injection was found to enhance cardiac function by increasing the amount of blood it can pump, resulting in a "dramatically improved survival rate."

After therapy, the pigs' hearts appeared to be less dilated and thinned out, resembling non-failing hearts.

The gene therapy used in the new study resulted in a 30% improvement in heart function, according to the researchers, while previous attempts only improved function by 5% to 10%.

The journal npj Regenerative Medicine published a study funded by the National Institutes of Health on Tuesday.

Despite the animals still experiencing stress on their hearts that can lead to heart failure, those who received treatment showed recovery of heart function and stabilization or shrinkage of the heart, according to TingTing Hong, MD, PhD, associate professor of pharmacology and toxicology at the University of Utah, in a release.

"This process is known as reverse remodeling, which involves restoring the heart to its normal appearance."

A new therapy to treat heart failure is being developed, according to Hong's statement to Planet Chronicle Digital.

"A possible new therapy to cure heart failure is on the way."

Hong was surprised to find that the gene therapy worked so well in large animals at such a low dose, the researchers discovered.

The "unprecedented" study marks a "new paradigm" for heart failure treatments, according to co-senior author Robin Shaw, MD, PhD, director of the Nora Eccles Harrison Cardiovascular Research and Training Institute at the University of Utah.

He informed Planet Chronicle Digital via email that the complex multi-organ syndrome of heart failure can be reduced to a treatable disease of failing heart muscle due to our treatment efficacy.

"Our low dose of gene therapy indicates that it will be safe for patients because the toxicity of gene therapy increases with dose."

According to Shaw, gene therapy, which has traditionally been used for rare diseases, could also be an effective approach for "acquired disease."

The study did have some limitations, the researchers acknowledged.

"Toxicology studies and dose escalation are necessary for the therapy to progress to the next stage, which is FDA approval," Hong stated.

Researchers are uncertain if gene therapy will be effective for individuals who have already acquired a natural immunity to the virus that carries the therapy.

The toxicology study is ongoing, and the team intends to commence human clinical trials in the fall of 2025, according to Hong.

Cardiologists weigh in

Dr. Jasdeep Dalawari, an interventional cardiologist and regional chief medical officer at VitalSolution, an Ingenovis Health Company in Ohio, did not participate in the research but expressed his thoughts on the findings.

"Gene therapy, precision medicine and personalized health care is the future."

The research conducted on animals is always intriguing, but it is crucial to test it on human subjects to determine if it will have the same impact in humans, he stated to Planet Chronicle Digital.

Injecting healthy genes is a similar intervention being explored for cystic fibrosis and muscular dystrophy, as genetic modifications are happening in various diseases.

"I am excited to discover more about the future of gene therapy, precision medicine, and personalized health care."

Dr. Johanna Contreras, an advanced heart failure and transplant cardiologist at the Mount Sinai Fuster Heart Hospital in New York City, pointed out that while conventional pharmacological interventions can ease cardiac stress and "systemic congestion," they primarily target the symptoms rather than the underlying cause of failing heart muscle.

Contreras stated that gene therapy could potentially change the expression of several proteins, which could subsequently affect the heart muscle's pathologic remodeling in heart failure.

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To determine the effectiveness of this therapy in humans and identify any potential side effects, Contreras emphasized the need for human trials beyond animal models.

"I eagerly anticipate gaining more knowledge and eventually determining if this could potentially benefit individuals with heart failure."