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. This is an acknowledged risk of
the treatment. The child had been successfully treated for X-SCID,
x-linked severe combined immunodeficiency, often known as ‘baby in the
bubble syndrome’, where boys are born with no immune system.
Professor Adrian Thrasher, and Professor Bobby Gaspar, consultant immunologists on the gene therapy programme, said:
“Our first thoughts are to secure the best treatment for this child and to support his family at this very difficult time.”
“The
gene therapy programme at Great Ormond Street Hospital/UCL Institute of
Child Health has treated ten children with X-SCID and five with
ada-SCID. All X-SCID children and most of the ada-SCID children have
seen clinical benefit. This unfortunate event is the first such
development on our programme.”
“As with any medical treatment
there are associated side effects. The development of leukaemia is now a
recognised side effect in this study, though the risks are balanced by
the severity of the condition and the lack of good alternative
treatments for X-SCID. This first study is now closed to recruitment
while safer improved formulations of the genetic medicine are being
prepared for clinical trials next year at several centres including
Great Ormond Street.”
“Every child matters. Families are
counselled very carefully before taking part in these treatments. Gene
therapy appears to offer a less intrusive treatment, for those patients
without a good bone marrow donor, and if we continue to make advances,
may become the treatment of choice. ”
“All patients are monitored carefully as part of their care plan.”
GOSH
has been working closely with the regulator, the Gene Therapy Advisory
Committee, (GTAC) since the discovery of the problem. Professor Martin
Gore, Chairman of GTAC is available for interview. He said today:
"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.”
“The
development of cutting-edge clinical trials at GOSH's world class gene
therapy centre continues to give much needed hope to children with fatal
immunodeficiency diseases. GOSH has been working hard to develop new
gene therapy vectors for future trials that should work better with
fewer side effects.”
“The data GTAC has seen from both the UK and
French trials suggests that the risk of leukaemia following gene
therapy may be confined to this patient group and the particular vectors
used in these 2 trials. The more that we understand about this specific
episode the better able we will be to develop safer and more effective
vectors.”
Contact information:
GOSH-ICH Press Office: 020 7239 3125
Email: Coxs@gosh.nhs.uk
For genuine and urgent out of hours call speak to switchboard on 020 7405 9200
