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Thursday, 3 October, 2002, 17:41 GMT 18:41 UK
Gene therapy trials halted
Rhys Evans
Rhys Evans underwent gene therapy in the UK in April
France and the United States have halted gene therapy trials after a child developed leukaemia after undergoing treatment.

The three year-old infant was being treated for potentially life-threatening "bubble boy" disease at a Paris clinic.

Authorities in France ordered a stop to trials as they started an investigation to see if the treatment triggered the leukaemia.

The other children are in good health

France's public health agency
That decision has prompted officials in the US to also suspend work on treating x-Severe Combined Immunodeficiency syndrome (x-SCID).

Advisors to the UK government decided against halting trials but have pledged to closely monitor treatments.

The infant was one of eight children being treated at Necker-Enfants Malades clinic in Paris for x-SCID.

Mutated gene

Children with x-SCID or "bubble boy" disease are born without an immune system. This is caused by a single mutated gene.

They must live in sterile conditions or risk picking up a life-threatening infection.

The treatment has been carried out successfully in the UK.

Earlier this year, doctors at Great Ormond Street Hospital in London cured 18-month-old Rhys Evans of the condition.

The three-year-old who has developed leukaemia received gene therapy in his first month of life.

The treatment was considered to have worked well. He had fought off an infection last spring that could have threatened his life.

Dr Alain Fishcher
Dr Fishcher expects the French trials to resume
However, in recent months his body started to produce too many white blood cells which is similar to children who develop leukaemia.

According to doctors, the boy is responding well to chemotherapy.

France's public health agency said treatment was halted as soon as the complications were discovered.

In a statement, it said: "The trials at the clinic have been suspended as a precaution until investigations into the causes have been completed."

It added: "The other children are in good health."

Dr Alain Fishcher, the lead scientists at the Paris clinic, said he was confident the trials would resume.

"The issue at stake is not to consider to do gene therapy or not to do gene therapy," he told the BBC.

"The question is whether we should or not modify the way we do gene therapy for that given diseases or the group of diseases we want to treat by gene therapy."

US action

In the US, officials said they had suspended trials there ahead of a meeting with authorities in Paris next week.

There are currently three trials underway in the US. One has already treated four children and two others were due to start shortly.

Patients who enrolled in a similar gene therapy study 10 years ago are now being contacted so that they can be advised of the risks.

The technique has also been used in Italy.

The decision to halt trials is a major blow to genetic therapy medicine.

The treatment of children with x-SCID has been one of the few successes.

The x-SCID trials involve taking a sample of bone marrow cells and deliberately infecting them with a retrovirus modified to carry the corrective gene.

The retrovirus - from the same virus family as HIV - inserts the therapeutic DNA randomly into the cells' chromosomes.

Hopefully, when the cells are returned to the patient, they are able to initiate the creation of a functioning immune system.

See also:

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