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GOSH announces leukaemia case following gene therapy for X-SCID

18 December 2007

We are sorry to announce that a child on the gene therapy programme at Great Ormond Street Hospital for Children NHS Trust has developed leukaemia, two years after treatment. This is an acknowledged risk of the treatment. The child had been successfully treated for X-SCID, x-linked severe combined immunodeficiency, often known as ‘baby in the bubble syndrome’, where boys are born with no immune system.

Professor Adrian Thrasher, and Professor Bobby Gaspar, consultant immunologists on the gene therapy programme, said:

“Our first thoughts are to secure the best treatment for this child and to support his family at this very difficult time.”

“The gene therapy programme at Great Ormond Street Hospital/UCL Institute of Child Health has treated ten children with X-SCID and five with ada-SCID. All X-SCID children and most of the ada-SCID children have seen clinical benefit. This unfortunate event is the first such development on our programme.”

“As with any medical treatment there are associated side effects. The development of leukaemia is now a recognised side effect in this study, though the risks are balanced by the severity of the condition and the lack of good alternative treatments for X-SCID. This first study is now closed to recruitment while safer improved formulations of the genetic medicine are being prepared for clinical trials next year at several centres including Great Ormond Street.”

 “Every child matters. Families are counselled very carefully before taking part in these treatments. Gene therapy appears to offer a less intrusive treatment, for those patients without a good bone marrow donor, and if we continue to make advances, may become the treatment of choice. ”

 “All patients are monitored carefully as part of their care plan.”

GOSH has been working closely with the regulator, the Gene Therapy Advisory Committee, (GTAC) since the discovery of the problem. Professor Martin Gore, Chairman of GTAC is available for interview. He said today:

"My sympathy goes out to the child who has developed leukaemia following gene therapy for X-linked SCID and their family. I also feel for the nurses, doctors and researchers in the gene therapy team at GOSH who are Utterly dedicated to helping children with serious life-threatening diseases. They are a highly regarded and professional group who have counselled families extensively about the risks of gene therapy, including the possibility of the development of leukaemia. I know that this child is in good hands and that colleagues at GOSH are doing everything possible to treat this child's leukaemia successfully.”

“The development of cutting-edge clinical trials at GOSH's world class gene therapy centre continues to give much needed hope to children with fatal immunodeficiency diseases. GOSH has been working hard to develop new gene therapy vectors for future trials that should work better with fewer side effects.”

“The data GTAC has seen from both the UK and French trials suggests that the risk of leukaemia following gene therapy may be confined to this patient group and the particular vectors used in these 2 trials. The more that we understand about this specific episode the better able we will be to develop safer and more effective vectors.”

Contact information:

GOSH-ICH Press Office: 020 7239 3125
Email: Coxs@gosh.nhs.uk
For genuine and urgent out of hours call speak to switchboard on 020 7405 9200

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 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.

Our charity needs to raise £50 million every year to help rebuild and refurbish Great Ormond Street Hospital, buy vital equipment and fund pioneering research. With your help we provide world class care to our very ill children and their families.