Posted on: 3 October 2002, source: New Scientist
A "miracle" gene therapy treatment for children suffering from the fatal "bubble boy" disease has been halted in France, after one of the patients developed leukaemia as a direct consequence of the treatment. However, British doctors argue that without the treatment many of the patients are certain to die, and say a similar trial in UK will continue. Boys with X-SCID (Severe Combined Immunodeficiency) have a faulty copy of a gene on their X chromosome that makes an immune protein called interleukin-2. As a result, they have no resistance to infection and die unless treated. In 2000, a team led by Alain Fischer at Necker Hospital, Paris, carried out the first gene therapy treatment, which replaced the faulty gene. It was one of only a handful of successful gene therapy trials in people. In April 2002, the mother of a Welsh boy treated at Great Ormond Street hospital in London described his progress as "nothing short of a miracle".