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New test for heart gene therapy

Tuesday August 12th, 2014

A patient with advanced heart failure has become the first person to test a gene therapy aimed at reinvigorating the heart, it has been announced.

Researchers in London, UK, are testing the treatment on patients with heart pumps - also known as left ventricular assist devices.

No more than 150 people are currently using these pumps, which are fitted to help patients survive until a heart transplant can be arranged.

The patient, Lee Adams, undergoing treatment at Harefield Hospital, London, is the first of 16, who will be invited to test the treatment.

Another eight people taking part in the research will be given placebo treatment.

Doctors also plan to test the effectiveness of the therapy by taking samples of heart muscle after six months to see if the gene is detectable and working.

The treatment aims to increase the levels of a protein SERCA2ain the heart muscle. Genes are delivered using a deactivated virus.

The trial at Imperial College, London, will also test the effectiveness of the treatment on patients who have previously been exposed to the virus and have developed antibodies that might neutralise it.

The gene therapy is already under trial on patients with less advanced heart failure.

Researcher Professor Sian Harding 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."

Mr Adams, aged 37, from Hertfordshire, UK, said: "Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a 'miracle cure' for myself and other patients. But I'm not building up my hopes too much because, for all I know, I might have had the placebo.

"If it does prove to be successful it would be exciting for patients who need a transplant but end up on the waiting list for a long time because of the shortage of donors."

The medical director of the British Heart Foundation, Professor Peter Weissberg, said: "Despite major advances in treating heart attacks, we’re still some way off a treatment that restores function in hearts damaged by one. This cutting-edge trial offers genuine hope of an effective treatment in the near future."

Tags: Genetics | Heart Health | UK News

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