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Second success for UK gene therapy trial

21 July 2003
By BioNews
Appeared in BioNews 217

A second patient in a gene therapy trial taking place at Great Ormond Street Hospital in London has been successfully treated, his parents and doctors announced last week. Christopher Reid, now two years old, received gene therapy for the immune system disorder X-linked severe combined immune deficiency (X-SCID) in December 2001. His treatment followed that of toddler Rhys Evans, whose recovery from the life-threatening illness was announced in April 2002.

Children affected by X-SCID have a faulty gene that means they have no working immune system, so their bodies cannot fight infections. Unless they can be given a matched bone marrow transplant, children with X-SCID face a lifetime living in a sterile bubble. 'Without gene therapy Christopher would not have lived to see his first birthday' said Rachel Reid, Christopher's mother. 'Even if we had decided to opt for a bone marrow transplant he could have become a casualty'. To treat Rhys and Christopher, the gene therapy researchers, lead by consultant paediatric immunologist Adrian Thrasher, first harvested bone marrow from the boys. They then isolated white blood stem cells from the bone marrow, which they infected with a virus carrying a working gene, before returning the cells to the boys' bodies.

Blood samples taken from Christopher last week revealed that his immune system is now working perfectly. 'The gene therapy has now restored most of his immune system, he takes protective drugs but can visit soft play areas and mother and toddler groups' said a hospital spokesperson. Rachel said her son has gone from being 'an unsettled, miserable baby into a happy confident toddler with a wonderful sense of humour'.

Christopher's parents decided to go public because of their concern about the 'negative coverage' of gene therapy over recent months. Concerns over the safety of the technique were raised earlier this year, when a second child in an X-SCID gene therapy trial at the Necker Hospital in Paris developed leukaemia. Investigations revealed that the virus used to deliver the therapeutic gene had inserted itself into or close to another gene called LMO2, previously linked to leukaemia. Similar trials in several countries were temporarily suspended, but restarted recently in both the UK and US. After weighing up the benefits and risks of the treatment, the UK's Gene Therapy Advisory Committee (GTAC) decided that 'it would be unethical to withdraw its approval of the UK X-SCID study'. The Great Ormond Street Hospital trial is funded by the charity 'Jeans for Genes', which runs an annual appeal to raise money for children affected by genetic disorders. The next 'Jeans for Genes' day will be held on 3 October 2003.

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Jeans for Genes announces second gene therapy success
Jeans for Genes |  17 July 2003
15 November 2010 - by Chris Chatterton 
Doctors from Great Ormond Street Hospital (GOSH) have used stem cells from donated cord blood to treat a child with a rare genetic disorder....
2 February 2009 - by Dr Megan Allyse 
Researchers in Italy and Israel have announced that they have successfully used gene therapy to treat ten children who suffer from a rare form of severe combined immunodeficiency (SCID) called ADA-SCID. The trial marks one of the first successful uses of gene therapy since past trials of...
27 April 2006 - by BioNews 
Replacement genes used to treat an inherited immune disorder could trigger cancer, new research carried out on rodents suggests. The study, published in the journal Nature, shows that treating mice with a normal copy of the gene defective in X-linked severe combined immune deficiency (X-SCID) results in cancer of the...
8 April 2005 - by BioNews 
A gene therapy technique that repairs faulty genes by 'editing' their DNA could lead to new treatments for inherited blood disorders and HIV, say US researchers. The approach could help address safety concerns over current gene therapy methods, many of which rely on specially adapted viruses to deliver working genes...
13 March 2005 - by BioNews 
Doctors at Great Ormond Street Hospital (GOSH) for Children and the Institute of Child Health have successfully treated a second disease using pioneering gene therapy treatment. The four-year old boy who received the therapy was affected by ada-SCID, a life-threatening immune deficiency condition. He is now attending pre-school and living...
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