Pioneering new injection to cure heart failure without need for major surgery
The technique, which involves a simple injection, could aid the recovery of hundreds of thousands of heart failure patients – and could even consign heart transplants to history.
A pioneering new treatment that allows damaged hearts to recover without the
need for major surgery is being trialled by one of Britain’s leading medical
The technique, which involves a simple injection, could aid the recovery of
hundreds of thousands of heart failure patients. Heart transplants could even be
consigned to history thanks to a trial by Imperial College, London, which aims to
show for the first time that gene therapy could repair failing organs.
Almost 500,000 people in Britain suffer from heart failure, with hundreds of
patients requiring new hearts. Around 200 transplants take place in Britain each
year. But two out of 10 people will die awaiting an organ due to severe
shortages of donors.
The trial involves researchers introducing a gene, created in a laboratory, into
heart failure patients to boost the production of a key protein, which they believe
will allow the muscle to recover.
Researchers say the treatment could offer a “viable alternative” to
transplantation. The British Heart Foundation, which is funding the trial, said the
discovery “offers genuine hope of an effective treatment in the near future”.
Dr Nick Banner, the consultant cardiologist at Harefield Hospital in north London,
who carried out the first infusion of the new gene therapy, said: “Advanced
heart failure is a progressive condition that results in a poor quality of life and
shortened life expectancy.
“The best treatment currently available is a heart transplant but the shortage of
donor organs in the UK means that many patients will die on the transplant
waiting list. Mechanical pumps can keep some patients alive long enough for a
donor heart to become available.
“The rationale for this study is to investigate the effectiveness of a new form of
therapy, which might in the future be a viable alternative to transplantation.
“This study will help us better understand whether the concept of repairing a
heart with gene therapy might be possible, even in patients with advanced heart
failure.” Heart failure usually occurs after damage or disease, and causes the
heart to become progressively weaker at pumping blood as its cells become
overstressed and begin to malfunction.
It can be brought on by high blood pressure; damage to arteries caused by
alcohol or smoking; weak heart muscles caused by genetic defects; or
In many cases heart failure is caused by damage after a heart attack, where
heart muscle and other tissue dies because its blood supply is cut off.
Some patients with advanced heart failure are fitted with a Left Ventricular
Assist Device (LVAD), a mechanical pump that supports the failing heart and
aims to restore normal blood flow.
The pump moves the blood from the left ventricle into the main artery (the
aorta) so it can circulate the oxygen-rich blood to the rest of the body.
Currently there are about 100 to 150 people in the UK living with a pump but
they are heavy and cumbersome and patients struggle to bathe or sleep while
wearing the battery packs.
The new therapy is designed to increase levels of SERCA2a, a protein in heart
muscle cells that plays an important role in heart muscle contraction.
In the new treatment, genes are pumped into the heart muscle cells to increase
the level of SERCA2a using a harmless engineered virus that will spread in the
organ and help repair the damaged muscle so it can pump on its own.
Previous studies have shown that the technique works in animals and on hearts
in a laboratory. Now human trials are to begin.
The team plans to 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.
Of the 24 patients enrolled in the study, 16 will be treated with the gene therapy
and eight will be treated with a placebo.
“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,” said Sian Harding, professor of cardiac pharmacology
and head of the British Heart Foundation’s Centre of Regenerative Medicine at
Imperial College London.
“It’s important to remember that the therapy is not correcting a gene defect,”
Prof Harding added.
“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.”
Prof Peter Weissberg, Medical Director at the BHF, said: “Heart failure
devastates the lives of hundreds of thousands of people in the UK. 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.”
Þ A drug taken by hundreds of thousands of Britons suffering from heart
arrhythmia may increase death rates, research has suggested. A study of more
than 122,000 Americans with atrial fibrillation showed that those given digoxin
were 20 per cent more likely to die than patients receiving different treatments.
About 800,000 people in Britain have AF, the most common type of arrhythmia
and the NHS prescribes around 5 million doses of digoxin each year.
The research, carried out by Stanford University Medical Center, was published
in the Journal of the American College of Cardiology.