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'Bubble boy' has leukaemia

18/12/2007

A child being treated for a condition known as 'baby in a bubble' syndrome has developed leukaemia, doctors treating him have confirmed.

Great Ormond Street Hospital (GOSH) said the young boy developed the illness two years into a gene therapy programme for the condition X-SCID, where baby boys are born with no immune system.

So far the hospital has treated ten children with the condition under the programme, with the case of leukaemia, a recognised side effect of the treatment, being the first to occur.

A similar treatment programme in Paris saw four out of 11 children with X-SCID develop leukaemia, three of whom recovered while one died.

Doctors say tests have shown the Paris leukaemia cases and today's confirmed case were caused by the gene therapy.

The cutting-edge treatment 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.

Modified cells then grow and ensure the child's immune function returns, sometimes completely.

But leukaemia can be a side effect as the introduced gene is implanted next to, and switched on, an oncogene - a cancer-causing gene.

The conventional treatment is bone marrow transplantation which has excellent results with a fully-matched donor, but less satisfactory results with a poorer match.

It also involves powerful chemotherapy and up to 20 per cent of children transplanted with a less than full match die.

"My sympathy goes out to the child who has developed leukaemia following gene therapy for X-linked SCID and their family," said Professor Martin Gore, chair of the regulatory body Gene Therapy Advisory Committee.

"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."

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

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