Page URL:

Second success for UK gene therapy researchers

13 March 2005
By BioNews
Appeared in BioNews 299

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 a life 'largely free of restrictions', say the team, who announced their success to coincide with the official opening of the new Wolfson Centre for Gene Therapy of Childhood Disease.

Mustaf, whose second name was not released, was diagnosed with ADA-SCID shortly after his birth, in September 2000. Children affected by SCID (severe combined immune deficiency) have a faulty gene that means their immune systems do not work properly, so their bodies cannot fight infections effectively. Some patients can be treated with a matched bone marrow transplant, but no suitable donor was available for Mustaf. Instead, he received regular enzyme replacement therapy - regular doses of the enzyme adenosine deaminase (ADA), which is missing in ADA-SCID patients. In December 2003 he became the first ADA-SCID patient at GOSH to be offered gene therapy. Doctors took a sample of his bone marrow, and used a harmless virus to deliver a working gene to his blood stem cells, which they then returned to his body. They also had to give Mustaf a mild dose of chemotherapy before the gene treatment, to kill off his existing bone marrow cells and make 'space' for the treated cells to grow and proliferate.

After spending five weeks in isolation, Mustaf was allowed to go home, although he is still receiving antibiotics and antibodies to bolster his immunity. However, these will shortly be withdrawn if his own immune system continues to develop, say the doctors. 'He has made steady progress and his immune system is already better than it was with enzyme replacement therapy', said team leader Adrian Thrasher. The trial was partly financed by the charity Jeans for Genes, which holds an annual fundraising day to raise money for research into new treatments for genetic conditions. The Department of Health, the Primary Immunodeficiency Association and the Child Health Research Action Trust also supported the trial.

Mustaf is the first ADA-SCID patient to be treated using gene therapy in the UK, although five patients have been successfully treated in Italy. The GOSH team has also used gene therapy to treat seven children and one adult affected by another immune disease, called X-SCID (X-linked severe combined immunodeficiency). The good news follows recent concerns over the safety of some gene therapy treatments, after a third child in a French X-SCID trial developed leukaemia. Critics say that gene therapy has failed to live up to its promise as a revolutionary new medical treatment. But Thrasher says that the time taken for gene therapy to produce successes is 'no different to that of other significant advances'. He also pointed out that 'unrealistic expectation has overshadowed genuine advances, and has focused attention more on clinical failures and unnecessary mistakes'.

British first as new therapy gives boy, 4, hope of normal life
The Times |  11 March 2005
British success heralds gene therapy revolution
The Daily Telegraph |  11 March 2005
Genes help immunity disease boy
BBC News Online |  10 March 2005
Second disease cured by gene therapy
Institute of Child Health |  10 March 2005
5 March 2012 - by Ayesha Jadoon 
Early trials in patients with chronic granulomatous disorder (x-CGD), a recessive X-linked condition that affects the immune system, have shown signs of success following the use of gene therapy. The observed boost in immunity of those undergoing treatment was only temporary, however, and the faulty gene causing x-CGD was not corrected permanently....
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...
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...
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...
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...
3 March 2003 - by BioNews 
Most US gene therapy trials that use viruses to deliver genes to bone marrow cells should be allowed to continue, Food and Drug Administration (FDA) advisors decided at a meeting held last Friday. Gene therapy trials for X-linked severe combined immune deficiency (X-SCID) were suspended in the US and several...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.