Gene hope for heart patients

Ben Hirschler

Tuesday, August 19, 2014

US biotech firm Celladon hopes that its Mydicar treatment can help Lee Adams and patients like him who have advanced heart failure and rely on devices to keep them alive until a donor heart becomes available.

The treatment works by inserting a gene called SERCA2a - the lack of which makes hearts pump weakly - directly into heart cells via a catheter to repair them. The trial will evaluate how much of the gene is getting to the heart muscle and how well it is working.

Adams is the first of 24 patients who will be given either the gene therapy or a placebo as part of a clinical study partially funded by the British Heart Foundation and sponsored by Imperial College London.

"Advanced heart failure is a progressive condition that results in a poor quality of life and shortened life expectancy," said Nick Banner, the consultant cardiologist at Harefield Hospital who carried out the first infusion. "The best treatment currently available is a heart transplant but the shortage of donor organs means that many patients will die on the waiting list."

Adams has been living for more than 2 years with a Left Ventricular Assist Device, which must always be connected to an external power supply via a lead through his abdomen.

"You can't just jump in the shower ... and it's difficult sleeping whilst being attached to it. Everywhere I go I have to carry the power supply and spare batteries in a backpack," he said.

The study complements an ongoing 250-patient trial, also funded by Celladon, which is investigating the benefits of gene therapy in 250 people with less advanced heart failure in Europe and the United States.

REUTERS

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Gene hope for heart patients