Gene targeting reverses cardiac fibrosis in heart-failure models

Mount Sinai's Roger Hajjar

The matricellular protein CCN5 can reverse cardiac scarring in animal models of heart failure, giving researchers a potential new drug target in the field, according to a new study published this week in the Journal of American College of Cardiology.

Researchers used trichrome staining and analysis of myofibroblast contents before and after CCN5 gene transfer to clearly show the reversal. Collectively, these data showed that CCN5 could potentially be used for the development of new anti-cardiac fibrosis therapies.

Cardiac fibrosis occurs when healthy cardiac cells are replaced with fibrous connective tissue, causing scarring and a stiffer cardiac muscle. It's known to be a key factor behind heart failure, which kills around 450,000 U.S. patients every year. 

While there currently are no effective cardiac fibrosis therapies available, it is considered a valid target for treatment. And though very early stage, this research may prove the start of a new interest for biopharma research for the disorder. 

"Our research is the first to demonstrate the ability to reverse cardiac fibrosis in heart failure models by targeting a specific gene," explained co-author Dr. Roger Hajjar, a professor of medicine and director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai. 

Dr. Hajjar and his team have already established that CCN5 is significantly lower in the myocardium of patients with severe heart failure.

He went on: "These findings demonstrate that CCN5 may provide a novel platform for the development of targeted anti-cardiac fibrosis therapies, which could benefit many patients with previously untreatable heart failure. At our Cardiovascular Research Center, we are dedicated to making significant strides in developing potential bench-to-bedside treatments for heart failure and other cardiovascular diseases."

The research, which is being funded from grants from the NIH and the Korean government, will continue to be investigated in preclinical models of heart failure with extensive fibrosis.

- check out the release
- read the journal abstract

Sponsored By Paraza Pharma

Choosing the Right Pre-Clinical Collaborative Research Organization (CRO)

Learn about the key factors that are essential to creating a collaborative and productive working relationship to advance pre-clinical drug discovery programs.