Encouraging results of gene therapy for severe combined immunodeficiency

December 16, 2004

A UK study in this week's issue of THE LANCET provides further evidence that gene therapy can be effective in creating a functional immune system for infants with severe combined immunodeficiency (SCID).

SCID is a genetic disease which prevents the immune system--particularly the white blood cells--from functioning. This leaves individuals at high risk of infection, as documented many years ago by the patient, David Vetter, who lived in a 'bubble' for 12 years to prevent infection from the external environment.

In the latest study, 4 children with SCID had stem cells removed from their own bone marrow; the relevant 'good' gene was identified from bone-marrow cells and spliced into a leukaemia virus. This virus was then introduced back into the patients where the gene was replicated. Almost normal immune functioning became established in all 4 patients after the procedure.

Lead investigator Adrian Thrasher (Institute of Child Health, London) comments: "At last follow up, all patients were at home in normal family and social environments, without restrictions on activities or exposure."

An accompanying commentary (p 2155) by Marina Cavazzana-Calvo and Alain Fischer (Hôpital Necker-Enfants Malades, Paris, France) discusses all clinical research done in this field: 'Remarkably, 17 of these 18 patients had their immunodeficiencies corrected with clear and sustained clinical benefits. The data show that the results of this gene-therapy strategy are reproducible.'
Contact: Dr Adrian Thrasher, Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London WC1N 1EH;
T) 44-207-905-2289;

Dr Marina Cavazzana, Département de Biothérapie, Hôpital Necker-Enfants Malades, 75015 Paris, France; E) m.cavazzana@neck.ap-hop-paris.fr


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