29 April 2006
Helen Phillips
Magazine issue 2549
Three leukaemia cases that arose during a well-known trial to treat "bubble boy" disease may have been an
inevitable consequence of the treatment.
GENE therapy was heralded as a miracle cure for some genetic diseases until its reputation was tarnished by a series of human trials. Now it seems that three leukaemia cases that arose during a trial that began in 1999 to treat X-SCID or "bubble boy" disease may in fact have been an inevitable consequence of the treatment, not just a rare side effect.
The affected boys were among 11 children being treated for the rare immune disorder X-SCID at the Necker Hospital for Sick Children in Paris, France. They had been born with a genetic defect that blocks the development of white blood cells, so couldn't fight-off infection, and the treatment aimed to correct that by inserting a replacement gene, IL2RG, into their bone marrow.
The boys' leukaemia was blamed on the new gene being inserted close to a gene called LMO2, which controls cell growth and can contribute to ...
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