A BREAKTHROUGH stem cell treatment that promises the first effective cure for heart disease will be offered to British patients this year, The Sunday Telegraph can reveal.
Hundreds of thousands of people stand to eventually benefit after regulators approved a major trial of the regenerative drug capable of shrinking fatal scar tissue from a heart attack.
The "off-the-shelf " therapy, which can be harvested for thousands of recipients from a single donor, will begin being administered to patients at London's Royal Brompton Hospital in November.
It is being pioneered by Prof Stephen Westaby, one of Britain's pre-eminent surgeons, who described the approval as "bloody marvellous" for patients who currently have "very poor length PAUL and quality of life". Currently around 50,000 people a year suffer heart attacks so severe the organ becomes scarred and cannot effectively pump blood around the body. These make up more than half a million people living with heart failure, for which there is no cure.
Developed by Celixir, the Warwickshire-based biomedical firm, the treatment delivers stem cells into the organ via an injection which then regenerates the heart cells, restoring function. The drug, called Heartcel, has been tried on 11 heart failure patients in Greece in 2012 and 2013, none of whom were expected to live more than two years.
Subsequent results showed the stem cells had rendered the hearts 78 per cent scar-free, meaning six years on all 11 are still alive and far more active than they previously were.
This week's approval by the US Food and Drugs Administration (FDA) means a coordinated international trial can now begin. It will involve 250 European participants spread across the Royal Brompton and two other undisclosed hospitals in the UK and mainland Europe, as well as 250 patients at centres in the US. If the trial replicates the "astonishing" results seen in the first Greek cohort, Celixir could apply for permission to provide Heartcel widely in the UK by 2021, according to Ajan Reginald, the company's chief executive.
Prof Westaby, formerly of the John Radcliffe Hospital, Oxford, said: "It has taken a long time to get to this stage but getting approval ... is absolutely massive for these patients. What is unique about Heartcel is that we are taking cells from a healthy donor and giving them to an unhealthy recipient, whereas previously people have tried using the patient's own cells."
Prof Westaby began investigating stem cells after he noticed that scarring in children with abnormalities in the coronary artery disappeared over time, and suspected they were playing a role.
Heartcel takes bone marrow stem cells from a donor with no heart problems, making them into enough doses to treat thousands of patients.
During production it undergoes genetic engineering making it "immune privileged", meaning it is biologically acceptable to all recipients and will not be rejected.
Previous trials at Barts and Great Ormond Street hospitals in London gave patients their own stem cells but were met with "modest results", said Prof Westaby.
Patients in the forthcoming Royal Brompton Hospital trial will be injected with Heartcel during the surgery they would have undergone anyway, however the drug could in future be delivered intravenously. Mr Reginald told The Sunday Telegraph his company had held preliminary talks with the National Institute of Health and Care Excellence (Nice) about approving the drug for NHS use.
He said that because the scar-reducing effect was so pronounced, the trial required a relatively small number of patients, making the research and development process and ultimate sale price cheaper.
"Currently these patients don't get better, they get worse," he said.
"If you can shrink the scar by half it's reasonable to say these patients should have a very significant improvement in quality of life and longevity."
'These patients should have a very significant improvement in quality of life and longevity'