UK boy has leukemia after SCID gene therapy

A 3-year-old UK boy who was treated as an infant with gene therapy for severe combined immunodeficiency (X-SCID) has developed leukemia, a blood cell cancer. In a press release on Tuesday, December 18, 2007, doctors at Great Ormond Street Hospital (GOSH) stated, "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." There have been other cases of boys with SCID developing leukemia after gene therapy, but those were in France; this is the first case in the UK.

In the same press release, Professor Martin Gore, Chairman of the Gene Therapy Advisory Committee overseeing the study, 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.”