Imagine: an injection that can cure…heart failure? If successful, it’s a revolution: almost 600,000 lives could be saved every year in the United States alone.
This is what my friend (and former Reagan and Bush Director of Policy Planning) Jim Pinkerton calls “The Cure Strategy“: applying a Silicon Valley-like approach to eliminating the things that ail us through technological advance, instead of what we might call “the Congressional Budget Office Strategy,” seemingly infecting both parties, which focuses on cost containment and triage. After all, if everyone doesn’t die young, how will Social Security stay solvent?
These scientists get it. So maybe I’ll have that Big Mac after all.
Pioneering New Injection to Cure Heart Failure without Need for Major Surgery
by Sarah Knapton
August 12, 2014
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 institutions.
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 infections.
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.”