Page URL:

xFrench gene therapy success

22 April 2002
By BioNews
Appeared in BioNews 154

A report in last week's New England Journal of Medicine shows that children treated by gene therapy at the Necker Hospital for Sick Children in Paris, France, are still living normal lives after more than two years.

Four boys were successfully treated in the French study, all having the X-linked genetic condition severe combined immune deficiency (X-SCID). Boys affected by the life-threatening disorder have to live in sterile environments or 'bubbles' to avoid contracting any infections. X-SCID is the same condition that Rhys Evans, the toddler who was successfully treated with gene therapy at London's Great Ormond Street Hospital recently, suffered from.

The Necker study was originally reported in May 2000. To carry out the gene therapy treatment, the French researchers harvested bone marrow from the patients. They then isolated blood stem cells from the bone marrow, which they infected with a virus carrying a replacement gene. After three days of repeated gene transfers, the cells were transplanted back into the patients. The immune systems of two of the boys, then aged eight and eleven months, appeared to begin to function normally and they left their sterile environment to go home. Two other boys on the gene therapy program were later also allowed home.

According to the new report, the condition of the four boys has not deteriorated, leading some people to believe that these are the first humans to be cured by gene therapy. However, Marina Cavazzana-Calvo, from the Necker Hospital, remains cautious about the use of the word 'cured', saying 'all we can say without problem is that the effect is reproducible in other children, and lasts long-term'.

French research offers promise of gene therapy cure
The Washington Post |  18 April 2002
Gene therapy restores more babies' immune systems
Yahoo Daily News |  17 April 2002
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...
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...
Log in 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.