Page URL:

Gene therapy trial for immune disorder hailed a success

2 February 2009
Appeared in BioNews 493

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 another form of SCID were shut down due to incidences of cancer amongst participants.

ADA-SCID is a rare genetic disorder that leaves children deficient in an enzyme called adenosine deaminase (ADA) which allows the body to fight outside contagions. Children with ADA-SCID can be treated with twice-weekly shots of adenosine deaminase or a bone marrow transplant. But the medicine is expensive and marrow transplants require a compatible donor and don't always work.

There were no available bone marrow donors for the ten children enrolled in the gene therapy trial. Instead, researchers removed marrow cells from the patients themselves and used a retroviral vector to give the cells working copies of the gene for adenosine deaminase. Then they injected the cells back into the patients. In most cases, the therapy was accomplished before the children reached the age of two. Results of the trial are reported two to eight years later. All ten patients were still alive, but two needed further treatment.

Gene therapy had a contentious history. While once hailed as the future of medicine it was nearly abandoned as a treatment technique after several early clinical trials went badly wrong, resulting in the death of at least one patient in the US. An earlier French trial to treat the most common form of SCID (X-SCID) was shut down after it was found that some participants had contracted cancer.

Thus far, none of the participants in this most recent study show any signs of cancer, or any other health problems, as a direct result of the gene therapy treatment. According to Dr Donald Kohn, a SCID expert at Children's Hospital Los Angeles and the University of Southern California, scientists are investigating in an effort to understand why treatment for one form of SCID should result in cancer and not another.

A gene therapy comeback?
Nature |  29 January 2009
Gene therapy cures form of 'bubble boy disease'
The Associated Press |  29 January 2009
Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
New England Journal of Medicine |  29 January 2009
Gene Therapy Works for Rare Immune Disorder
MedPage Today |  28 January 2009
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....
30 August 2011 - by Rosemary Paxman 
Over a dozen children with 'boy in bubble' syndrome are alive and well, with functioning immune systems, nine years after undergoing gene therapy to correct their disorder, researchers report....
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....
7 January 2008 - by Stuart Scott 
A boy enrolled on a pioneering gene therapy trial has developed leukaemia, his doctors based at London's Great Ormond Street Hospital have announced. The three-year-old was of one of 10 patients treated for X-SCID: a genetic disorder whereby a mutation in the IL2RG gene leaves carriers without...
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...
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...
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...
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.