Cardiac gene therapy trial treats first patient

A Scottish patient has become the first person in the world to receive a pioneering therapy aimed at improving outcomes for those having specialist heart surgery.

Heart surgeons during a heart operation, wearing scrubs

The treatment involves precisely editing DNA in veins to be used during heart bypass surgery to boost the production of a protective protein.

The treatment could help extend the lifespan of blood vessels used during the surgery and significantly improve patient health, experts say.

Heart bypass

Heart bypass surgery – an operation to improve blood flow to the heart – is a life-saving treatment for patients with coronary heart disease.

The process typically uses one artery and two or more veins as bypass grafts – healthy blood vessels used to bypass a narrowed or blocked artery – creating a new route for blood to flow. 

Vein grafts used in this type of surgery can fail because they are not naturally designed to withstand the high pressure of blood flow from the heart.

Protect grafts

The PROTECT study, led by NHS Greater Glasgow and Clyde and the University of Glasgow in collaboration with NHS Golden Jubilee and the University of Edinburgh, is trialling a new gene therapy designed to support newly grafted blood vessels. 

The treatment will introduce a gene, which produces a protein called TIMP-3, into the vein to be grafted. 

TIMP-3 is involved in tissue remodelling. Higher levels of the protein could help to prevent thickening and blockage of the blood vessel over time, scientists say.

Exciting milestone

The research team has developed a way to treat the graft directly at the time of surgery, safely and efficiently delivering the gene therapy to the affected tissue before grafting into the heart.

It is hoped the treatment will help to extend a patient’s healthy life expectancy and reduce the need for further surgeries, experts say. 

The clinical trial is supported by the Medical Research Council and British Heart Foundation and has received additional support from the Cell and Gene Therapy Catapult, University of Glasgow Impact Accelerator Award and Northern Alliance Advanced Therapy Treatment Centre, as well as the Universities of Bristol and Cardiff. 

Our team has developed a new approach to prevent vein graft failure. The new gene therapy has been developed during more than two decades of teamwork involving many experts working in collaboration.

We are delighted to be leading this new study which is designed to clarify the feasibility and potential benefits of this new therapy for patients undergoing heart bypass surgery.

I’m hugely grateful to the Medical Research Council and British Heart Foundation for helping the team translate this laboratory discovery to clinical evaluation.

We have faced many challenges but our interdisciplinary team and funders working together have enabled the trial to become a reality.

This pioneering study is an inspiring reminder of how far gene therapy, which was once a distant scientific ambition, has come.

Research funding from the BHF to Professor Andrew Baker over many years laid the groundwork for this study, generating the knowledge, experience and proof of concept data needed to move this research out of the lab and towards the clinic.

In partnership with the MRC, continued BHF funding has enabled Professors Berry and Baker to begin the clinical trials necessary to prove the value of this new therapy. We look forward to seeing the results of this exciting trial in a few years.

Related links

Centre for Cardiovascular Science

NHS Glasgow and Greater Clyde announcement

Image credit: Westend61 via Getty Images

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