This is the start of a new clinical trial that will assess gene therapy for patients with heart pumps and provide detailed insight on its impact on the heart muscle.
Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the
The LVAD is an electrically driven pump, moving the blood from the left ventricle into the main artery (aorta) so it can circulate the oxygen-rich blood to the rest of the body. Individuals with advanced heart failure who require a transplant may be fitted with an LVAD to keep them alive until a suitable donor heart becomes available. Currently there are around 100 to 150 people in the
The new trial, led by
This particular gene therapy is designed to increase levels of SERCA2a protein in heart muscle cells. SERCA2a plays an important role in heart muscle contraction. Genes are inserted into the heart muscle cells to increase the level of SERCA2a using a harmless engineered virus that is based on a naturally occurring virus. In this study the research team will 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.
The research team plan to evaluate how this therapy works in 24 patients with advanced heart failure who are fitted with LVADs. Of the patients enrolled in the study, 16 will be treated with the gene therapy and eight will be treated with a placebo. Dr
"LVADs 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. The patients taking part in the trial will have regular tests to see if there are any improvements in heart function."
Lee is hopeful about the outcome but careful not to get too carried away. 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' 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 new study will be the first to evaluate whether gene therapy is delivered to the heart muscle and if its effectiveness is compromised in patients with antibodies to the virus, which delivers the gene.
Previously patients with an antibody to the naturally occurring version of the virus (about 50-60 per cent) have been excluded from a larger ongoing CUPID2 trial, which is investigating the benefits of gene therapy in people with heart failure but no LVAD device. Patients were excluded because it was believed possessing the antibody would render the virus less effective at inserting the gene into the heart muscle. However the actual effect of the antibody has never been explored in patients. This new study will evaluate the effectiveness of the gene therapy in both those patients who have the antibody and those patients without the antibody in order to make a comparison.
"We have adapted the wild virus by removing the viral genes and replacing them with the treatment
The 24 patients in the trial, recruited from
This trial complements the larger ongoing CUPID2 trial, also funded by
Keywords for this news article include: Antibodies, Biotechnology, Surgery, Viruses, Genetics, Hospital, Virology, Cardiology, Immunology, Gene Therapy, Cardio Device, Heart Disease, Heart Failure, Bioengineering, Blood Proteins, Immunoglobulins, Medical Devices, Heart Transplant, Organ Transplant, Cardiovascular Diseases,
Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC
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