British scientists have launched a pioneering trial to see if gene therapy could replace heart transplants.
By: Jo WilleyPublished: Tue, August 12, 2014
The therapy involves injecting a harmless altered virus into the blood stream [GETTY/PIC POSED BY MODEL]
Lee Adams, a 37-year-old carpenter, is the first patient in the world to take part in a gene therapy trial while wired up to a mechanical heart pump.
The study is to investigate the use of the therapy on 24 patients with advanced heart failure, recruited from Harefield Hospital, London, and Papworth Hospital, Cambridgeshire.
All the participants are kept alive by a Left Ventricular Assist Device while they wait for heart transplants.
Many such patients endure agonising delays in finding a suitable donor.
Sixteen randomly chosen patients will be treated with a corrective gene to help their hearts beat more strongly.
Eight others will receive a “dummy” placebo therapy.
Lee, from Rickmansworth, Herts does not know which group he is in.
He 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’.
No matter what the cause of the heart failure, the therapy should be equally beneficial for patients
Professor Sian Harding
“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 therapy involves injecting a harmless altered virus into the blood stream to carry the corrective gene into heart muscle cells.
The aim is to raise levels of a protein called SERCA2a, which plays a vital role in heart muscle contraction.
Six months after the treatment, biopsies of heart muscle will be taken to see if the gene is present and functioning.
The hearts of patients who subsequently have transplants will also be examined.
Professor Sian Harding, who helped develop the treatment at Imperial College London, said: “No matter what the cause of the heart failure, the therapy should be equally beneficial for patients.”
The trial complements a larger study, Cupid2, which is investigating the effectiveness of gene therapy in 250 US and European patients with less advanced heart failure.