First gene therapy trial launched for heart patients with mechanical pumps

PUBLIC RELEASE DATE:

11-Aug-2014

Contact: Franca Davenport f.davenport@imperial.ac.uk 44-207-594-3415 Imperial College London

For the first time in the world, a patient with a mechanical heart pump has taken part in a new gene therapy trial for heart failure at Harefield Hospital, London.

This is the start of a new clinical trial that will assess gene therapy for patients with heart pumps and provide detailed insight on its impact on the heart muscle.

Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the UK. Some individuals with an advanced heart failure can be fitted with a Left Ventricular Assist Device (LVAD), which supports the failing heart and aims to restore normal blood flow.

The LVAD is an electrically driven pump, moving the blood from the left ventricle into the main artery (aorta) so it can circulate the oxygen-rich blood to the rest of the body. Individuals with advanced heart failure who require a transplant may be fitted with an LVAD to keep them alive until a suitable donor heart becomes available. Currently there are around 100 to 150 people in the UK living with an LVAD.

The new trial, led by Imperial College London and funded by the British Heart Foundation (BHF) and Celladon Corporation, will explore whether this gene therapy could help these patients' hearts recover and potentially provide an alternative treatment. It is the first study of gene therapy in this patient group. The work was initiated with support from the Leducq Foundation.

This particular gene therapy is designed to increase levels of SERCA2a protein in heart muscle cells. SERCA2a plays an important role in heart muscle contraction. Genes are inserted into the heart muscle cells to increase the level of SERCA2a using a harmless engineered virus that is based on a naturally occurring virus. In this study the research team will take small biopsy samples of the heart muscle six months after treatment to measure if the gene is present, detectable and functional in the patients' hearts.

Professor Sian Harding, Professor of Cardiac Pharmacology and Head of the BHF Centre of Regenerative Medicine in Imperial College London, who helped develop the treatment, said: "We will be using state-of-the art methods to gain detailed information on how and where the gene therapy takes effect, which will potentially help us develop and improve the therapy. It's important to remember that the therapy is not correcting a gene defect. We are working much more downstream, which means that no matter what the cause of the heart failure, the therapy should be equally beneficial for patients whether their heart problems stem from genes, lifestyle or the environment or a mixture of all of these."

Original post:
First gene therapy trial launched for heart patients with mechanical pumps