http://bmj.com/cgi/content/full/325/7368/791/a
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The UK Department of Health's gene therapy advisory committee (GTAC) has this week recommended that additional measures be put in place to protect patients undergoing gene therapy trials.
This comes in response to the suspension of gene therapy trials at Necker Hospital, Paris, when a boy being treated for X linked severe combined immunodeficiency syndrome (SCID) developed leukaemia.
The committee was alerted to the case on 16 September and has subsequently carried out a review of ongoing gene therapy trials in the United Kingdom. Only two studies, both involving the treatment of children at the Hospital for Sick Children, Great Ormond Street, London, were identified as being closely related to the French study.
"GTAC considers this a most serious case," said Professor Norman Nevin, the committee's chairman. "Yet in balancing the potential risks and benefits to these children, and in weighing up alternative treatment options, we have decided that, at this time, it would be unjustifiable to withdraw GTAC's approval of the two Great Ormond Street studies."
Gene therapy for patients with X linked SCID, in which the patient's bone marrow cells are deliberately infected with a retrovirus modified to carry the corrective gene for a functioning immune system, was successfully used to treat an 18 month old boy, Rhys Evans, at Great Ormond Street earlier this year (13 April, p 872).
Professor Adrian Thrasher, consultant immunologist at Great Ormond Street, who was involved in the successful treatment of Rhys Evans, thought the risk of other patients' developing leukaemia was very low.
He told the BMJ: "Our studies are very similar [to the French study] and use similar viruses. The problem in France has arisen because the transgene was inserted close to an oncogene and has activated it... However, it is almost certain that the leukaemia would have needed additional triggers, for example genetic predisposition and viral infection, but these are much harder to determine."
According to Professor Thrasher, in the thousands of animal studies that have
used this type of technology a mutation of this kind has been
documented only once.
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Collections under
which this article appears:Genetics
Haematology
Incl Blood Transfusion |
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