In a European first, a little-known gland called the ‘thymus’, some of which is routinely removed during cardiac surgery, has saved the lives of children with a life threatening immunodeficiency condition, complete DiGeorge syndrome (cDGS).
Thymus tissue is removed as standard from children undergoing cardiac surgery for congenital heart defects in order to allow the surgeons to perform the heart procedure. This tissue is normally discarded. As part of the study, this tissue was grown in a specialist laboratory and then transplanted into children with cDGS.
Great Ormond Street Hospital (GOSH) is one of only two centres in the world developing this treatment. The study into this procedure was published today in The Journal of Allergy and Clinical Immunology.
The procedure left 75 per cent of the cDGS patients on the trial with working T cells – the ‘fighter cells’ vital to a fully-functioning immune system. Long term, these children should now have a vastly improved life expectancy and no longer be at risk of falling dangerously ill from common infections.
The lead researcher, GOSH Consultant Paediatric Immunologist, Professor Graham Davies said: “For these children, this procedure is a life saver. Without it, those born with cDGS are likely to die from infection before the age of two. This technique also has the potential to benefit children living with other severe immune system disorders – not just cDGS.”
Professor Graham Davies is also an Honorary Clinical Professor of Paediatric Immunology at UCL Great Ormond Street Institute of Child Health (ICH).
Professor David Goldblatt, Director of Research and Development, Great Ormond Street Hospital said: “The team worked closely with the families and cardiac colleagues to bring this pioneering procedure to the UK and Europe.
“Here at GOSH and the ICH we are committed to working in collaboration with our rare disease colleagues to continue to innovate and bring lifesaving treatments to children across the UK, and indeed further afield.”
Taking part in the trial
Aiden from Oldbury took part in the trial. His mum, Megan, says:
“Aiden was diagnosed with complete DiGeorge Syndrome when he was one. We were devastated when we heard the news. We’d never heard of the condition and suddenly we were being told he had virtually no immune system and probably wouldn’t live past three years old.
“For the next year he was confined to hospital and hooked up to a ventilator 24-hours a day. His future looked bleak.
“It was at this time that we were told about a thymus transplant trial at Great Ormond Street Hospital and knew it was our only hope. The procedure was relatively straightforward and within a year his health had dramatically improved.
“Now seven years old, Aiden is at school and loves playing tennis, reading Roald Dahl books and playing with his brother and sister.“He’s living a life we didn’t think he would have. Without the trial, we wouldn’t have Aiden. It’s changed our lives and saved his.”
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Notes to Editors
Great Ormond Street Hospital for Children NHS Trust is the country’s leading centre for treating sick children, with the widest range of specialists under one roof.
With the UCL Great Ormond Street Institute of Child Health, we are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.
Great Ormond Street Hospital Children’s Charity needs to raise money to support the hospital to give children who need help the most the best chance for life. The charity funds patient and family support programmes, provides the latest medical equipment and supports the essential redevelopment of the hospital. It has also launched a five-year strategy to support research in some of the most serious and complex childhood diseases. Great Ormond Street Hospital Children’s Charity is the largest dedicated funder of paediatric research in the UK and our work is entirely funded through the generosity of supporter donations. For more information visit www.gosh.org