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Scientists Discover Cause Of Leukaemia That Halted Treatment Trial For 'Baby-in-a-Bubble' Syndrome
October 16, 2003Medical Research Council (MRC) scientists have helped establish the cause of the leukaemia which developed in two young patients taking part in a pioneering gene therapy trial to treat the fatal 'baby-in-a-bubble' syndrome, severe combined immunodeficiency (X-SCID).
The trial at the Necker-Enfants Malades clinic in Paris was stopped last year due to the higher than expected number of cases of leukaemia amongst the eight children taking part. After three years of successful gene therapy for X-SCID, leukaemia occurred in the two youngest patients undergoing treatment.
Contributing to the international effort to establish quickly why the leukaemia developed, the scientists reveal in the journal Science that in the cases of the two boys, the gene therapy corrected the faulty gene but also activated a cancer-causing gene called LMO2.
The scientists suggest that a number of factors might explain why this occurred at a very much higher frequency than expected and that by modifying these factors, scientists will be able to deliver a gene therapy to treat X-SCID which has a much lower risk of causing leukaemia.
X-SCID is a genetic disease only found in boys. Those with X-SCID, or 'baby-in-a-bubble' syndrome, are born without an immune system forcing them to live in sterile conditions or risk picking up a life-threatening infection. The disease is caused by a single mutated gene and in the absence of a matched bone marrow donor there is no cure other than gene therapy.
Lead MRC researcher, Dr Terry Rabbitts of the MRC Laboratory of Molecular Biology, said:
"Cancer is a possible side effect of gene therapy, so any research which will help scientists to modify the treatment to reduce that risk is of great importance.
"Although it is anguishing for parents to expose their children to the chance of developing cancer, the benefits of gene therapy for this devastating disease greatly outweigh the risks of the disease itself as X-SCID can only be treated if a matched bone marrow donor is available."
Both children who developed leukaemia during the trial underwent treatment for their cancer with chemotherapy and bone marrow transplants. They are alive and well, and in complete clinical remission.
Up to one in 50,000 births in the UK are affected by X-SCID.
Dr Rabbitts worked on this project in collaboration with scientists from the Babraham Institute and across Europe and the USA.
ENDS
Medical Research Council
The scientists suggest that a number of factors might explain why this occurred at a very much higher frequency than expected and that by modifying these factors, scientists will be able to deliver a gene therapy to treat X-SCID which has a much lower risk of causing leukaemia.
X-SCID is a genetic disease only found in boys. Those with X-SCID, or 'baby-in-a-bubble' syndrome, are born without an immune system forcing them to live in sterile conditions or risk picking up a life-threatening infection. The disease is caused by a single mutated gene and in the absence of a matched bone marrow donor there is no cure other than gene therapy.
Lead MRC researcher, Dr Terry Rabbitts of the MRC Laboratory of Molecular Biology, said:
"Cancer is a possible side effect of gene therapy, so any research which will help scientists to modify the treatment to reduce that risk is of great importance.
"Although it is anguishing for parents to expose their children to the chance of developing cancer, the benefits of gene therapy for this devastating disease greatly outweigh the risks of the disease itself as X-SCID can only be treated if a matched bone marrow donor is available."
Both children who developed leukaemia during the trial underwent treatment for their cancer with chemotherapy and bone marrow transplants. They are alive and well, and in complete clinical remission.
Up to one in 50,000 births in the UK are affected by X-SCID.
Dr Rabbitts worked on this project in collaboration with scientists from the Babraham Institute and across Europe and the USA.
ENDS
Medical Research Council
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