Gene therapy falters

Possibility of leukemia-like side-effect halts trials

Brendan Maher(bmaher@the-scientist.com)
Oct 7, 2002

Health officials and clinicians have suspended gene therapy trials for Severe Combined Immune Deficiency (SCID) in the United States, France, and Germany following observation of a leukemia-like disorder in one patient who underwent the treatment. Recently hailed as gene therapy's first real success, the therapy had restored immune function to at least 14 children with X-linked SCID, the so-called "bubble boy" disease.

Alain Fischer at Necker Children's Hospital in Paris observed elevated T-lymphocyte levels initially thought to be associated with a chicken pox infection. The levels continued to rise after the infection subsided, and in September, Fischer contacted colleagues and health officials.

Christof von Kalle and colleagues at Cincinnati Children's Hospital are now trying to determine whether the condition, which Fischer calls lympho-proliferation, is caused solely by the insertion of the "therapeutic" gene into an inappropriate place in the genome. Cancer-causing insertions have long been a concern of those...

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