Gene Therapy Trial For Patients With Mechanical Heart Pumps Has Begun

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The first trial for a gene therapy drug for heart failure has begun

Last week, for the first time, a patient with a mechanical heart pump was administered an experimental gene therapy drug to prevent heart failure, in Harefield Hospital, London. This mark the beginning of a clinical trial that will assess the efficacy of a gene therapy drug for people who have mechanical heart pumps.

The trial is being funded by Celladon and the British Heart Foundation, and led by Imperial College London.

The gene therapy drug works by boosting the level of SEERCA2a protein present in the muscle cells of the heart. This protein is vital in enabling the heart muscle to contract. The gene therapy comes in the form of a genetically altered viral vector, and is inserted directly into the heart muscle. The inserted genes stimulate the translation of SERCA2a protein. After this procedure is carried out on patients, a biopsy of the heart muscle will be taken in order to determine if the relevant gene is present and being transcribed.

Professor Sian Harding at Imperial College London, who was part of the team that developed the drug, said that the team will be “…using state of the art technology to gain detailed information on how and where the gene therapy takes effect, which will potentially help us to 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 theseProfessor Sian Harding, Professor of Cardiac Pharmacology and Head of the BHF Centre of Regenerative Medicine at Imperial College London

The drug will be tested on 24 patients who have mechanical heart pumps and the trial will include a randomised double-blind placebo arm.

If successful, this gene therapy could radically improve the quality of life of patients afflicted with heart failure.

Read more about it here: First Gene Therapy Trial for Heart Patients with Mechanical Pumps

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