Potential gene therapy for chronic heart failure

10th May 2016

A potential gene therapy for treating chronic heart failure may soon start human trials.

The treatment, carfostin, is an experimental treatment from NanoCor Therapeutics.

It has just been accepted by the US Food and Drug Administration as an investigational new drug.

The company says this is an important milestone, and it intends to start a phase one trial later this year in people with advanced chronic heart failure.

Gene therapy is a technique that uses genes to treat or prevent disease. It can enable doctors to treat a disorder by correcting a faulty gene. Researchers are testing several approaches to gene therapy, including replacing a faulty gene with a healthy one, knocking out a faulty gene’s ability to cause disease, and introducing a new gene into the body to help fight a disease.

Carfostin is an intracellular protein therapy, delivering a therapeutic gene, protein phosphatase-1 Inhibitor-1. It is hoped the treatment will not only improve the heart’s ability to contract but will also help reduce the heart’s enlargement to a more normal size.

Dr Roger Hajjar, NanoCor scientific co-founder and director of the cardiovascular research centre at Icahn School of Medicine in New York, said: “Gene therapy is a viable option for the treatment of chronic heart failure, and our proprietary technology platform allows for targeted and minimally-invasive delivery of the therapeutic to the heart."

For more details, see here