BRITISH scientists are stepping up clinical tests of gene therapy in a bid to help people with advanced heart failure pump blood more efficiently.

Researchers say they plan to enrol patients into two new clinical trials using Mydicar, a gene therapy treatment made by privately held US biotech company Celladon.

After more than 20 years of research, the groundbreaking method for fixing faulty genes is starting to deliver, with European authorities approving the first gene therapy for rare metabolic disease last November.

In the case of heart failure, the aim is to insert a gene called Serca2a directly into heart cells using a modified virus, delivered via a catheter infusion. Lack of Serca2a leads to ever weaker pumping in people with heart failure.

Although drugs offer some relief, there is currently no way of restoring heart function and the prognosis for those with advanced disease is worse than for many cancers.

One of the studies, led by scientists at Imperial College London, is part of a wider midstage phase 2 project sponsored by Celladon that involves 200 patients worldwide, some of whom have already been treated in the US and Denmark.

The second trial, which is due to start in July, will test the same treatment in 24 British patients already fitted with mechanical heart pumps to see how the approach may help in this particular setting.

It promises to be a long haul, with extensive phase 3 studies still needed once results of the current mid-stage tests are received, which Celladon expects in the first half of 2015.

Gene therapy has experienced a series of advances and setbacks over the decades. The most notable blow came in 1999 when an Arizona teenager died in a gene therapy experiment. More recent results, however, have been promising in fields ranging from immune system diseases to blindness.

"It is a great example of the slow burn of good laboratory science translating into a potential clinical treatment," says Dr Peter Weissberg, medical director of the British Heart Foundation, which is co-funding the second trial.

Original post:
Another piece in the puzzle of how to heal broken hearts

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