First Gene Therapy Trial for Heart Patients with Mechanical Pumps

For the first time in the world, a patient with a mechanical heart pump has received a new gene therapy 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.

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 the 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."