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More successes for gene therapy treatment

17 December 2004
By BioNews
Appeared in BioNews 289

Four children who received gene therapy for a severe inherited immune disorder are now all at home living normal lives, UK scientists report. The boys, who were affected by severe combined immunodeficiency (SCID), received the pioneering treatment at Great Ormond Street Hospital in London. In a commentary accompanying the study, which is published in the Lancet, French gene therapy researchers Marina Cavazzana-Calvo and Alain Fischer say that 17 SCID patients have now been successfully treated in this way.

Children affected by SCID have a faulty gene that means they have no working immune system, so their bodies cannot fight infections. This life-threatening condition is sometimes called 'bubble boy' disease, as unless they can be successfully treated with a matched bone marrow transplant, patients must spend their lives in a sterile environment. To carry out gene therapy treatment, doctors must first harvest bone marrow from the patients, from which they isolate blood stem cells. They then infect these cells with a retrovirus (a virus that inserts its genetic material into the host cell's DNA) carrying a working gene, before returning the modified cells back to the patients.

A team lead by Fischer, at the Necker Hospital in Paris, announced the first successful use of gene therapy to treat SCID in 2000. Then, in 2002, toddler Rhys Evans became the first UK patient to benefit from the new approach. However, the field suffered a serious setback later that year, when it emerged that two of the French patients developed leukaemia as a result of the treatment. Although both responded well to treatment for the leukaemia, governments in many countries temporarily suspended similar gene therapy trials, while the exact cause of the cancer was determined.

No more patients in either the UK or French trials have developed leukaemia. In their Lancet commentary, Fischer and Cavazzana-Calvo discuss all of the SCID clinical research done so far, and conclude that '17 of these 18 patients had their immunodeficiencies corrected with clear and sustained clinical benefits'. They added that 'the data show that the results of this gene therapy strategy are reproducible'. Great Ormond Street team member Bobby Gaspar told BBC News Online that the results could pave the way for developing gene therapy treatments for other inherited immunodeficiencies, as well as blood disorders such as sickle cell anaemia.

Boys 'cured' with gene therapy
BBC News Online |  17 December 2004
Encouraging Results of Gene Therapy for Severe Combined Immunodeficiency |  16 December 2004
Success rate grows for gene therapy to treat "bubble" children
Yahoo Daily News |  16 December 2004
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...
7 March 2005 - by BioNews 
The US Food and Drug Administration (FDA) has recommended that gene therapy treatments for an inherited immune disorder are limited to those who have no alternative. The move follows news that a third child in a similar French trial has developed leukaemia. The trial for X-linked severe combined immunodeficiency disorder...
31 January 2005 - by BioNews 
A gene therapy trial for an inherited immune deficiency disorder has been suspended again, following the appearance of complications in a third child. Eleven patients affected by X-linked severe combined immunodeficiency disorder (X-SCID) have so far been treated by the team, based at the Necker Hospital in Paris. While most...
14 June 2004 - by BioNews 
A French gene therapy trial halted after two participants developed leukaemia looks set to restart after a 22-month suspension, reports Nature. Scientists at the Necker hospital in Paris successfully treated nine children with an inherited immune system disorder called X-linked severe combined immune deficiency (X-SCID). But the trial, along...
17 October 2003 - by BioNews 
US and European scientists have published results confirming that a gene therapy technique triggered leukaemia in two boys taking part in a trial at the Necker hospital, Paris. Their findings, which appear in Science, show that the virus used to deliver the therapeutic gene activated a cancer-causing gene. Eleven boys...
21 July 2003 - by BioNews 
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...
16 December 2002 - by BioNews 
US gene therapy trials for a rare immune disorder, suspended after a patient in a similar French trial developed a leukaemia-like disease, should now go ahead, says the Recombinant DNA Advisory Committee (RAC). In October, gene therapy trials for severe combined immunodeficiency (SCID) were halted in several countries, after scientists...
7 October 2002 - by BioNews 
Gene therapy trials taking place in France and the US were suspended last week after a boy being treated at the Paris Necker Hospital developed leukaemia-like symptoms following treatment. French public health officials ordered a halt to the trials while they undertook an investigation and the decision was quickly mirrored...
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