‘Baby in a bubble’ pioneer has leukaemia

‘Baby in a bubble’ pioneer has leukaemia

Last Updated:4:26pm GMT 18/12/2007

A child undergoing pioneering gene therapy for what has become known as “baby in the bubble syndrome” has developed leukaemia.

Great Ormond Street Hospital has announced that the boy, who was born with no immune system, has developed the chronic bone marrow disease two years after successful treatment for X-SCID.

Affected children produce no lymphocytes and have no natural defence against infection, meaning they must be kept isolated from the outside world.

X-SCID affects only boys and is caused by a single faulty gene.

In the first trial of its kind in Britain, 10 babies with two types of potentially fatal immune system diseases were given the treatment.

Medics in the UK knew that there was a risk of the children developing leukaemia as a result of the treatment, but the boy, who is three years old, has been the first to do so.

A trial in France of a similar therapy was stopped in 2002 after four of eleven children developed leukaemia.

Professor Adrian Thrasher and Professor Bobby Gaspar, consultant immunologists on the gene therapy programme, said in a statement: “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.”

They added: “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.”

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

Affected children are likely to die within a year without a bone marrow transplant.

Gene therapy involves a working copy of the defective gene being placed in the child’s bone marrow cells and these are then returned to the child.

Great Ormond Street has been working with the regulator, the Gene Therapy Advisory Committee (GTAC), since the discovery of the boy’s condition at the end of last month.

Professor Martin Gore, GTAC chairman, said: “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.”

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