A type of gene therapy helps restore a working immune system to children with a genetic disease called x-linked severe combine immune deficiency (SCID-X1), which raises the risk of serious infections, new research shows.
In fact, preventive medications, such as antibiotics, could be stopped for two of the four young children included in the study, Dr. Adrian J. Thrasher, at University College London, and his team report in The Lancet medical journal.
The therapy works by delivering a normal copy of gene that is mutated in kids with SCID-X1.
This approach helped restore various immune cells to normal or near-normal levels. At last follow-up, between 12 and 29 months after the treatment, “all patients were at home in normal family and social environments, without restriction on activities or exposure,” reports Thrasher’s team.
With refinements in technique, “there is potential to establish gene therapy as a standard therapeutic procedure for this type of disease,” the authors note.
In a related commentary, Drs. Marina Cavazzana-Calvo and Alain Fischer, from Hopital Necker-Enfants Malades in Paris, note that altogether, there have now been 18 cases of SCID that have been treated with this type of gene therapy, 17 of whom derived “clear and sustained clinical benefits.”
The current findings coupled with other promising reports “should encourage researchers to extend the treatment to target other life-threatening immunodeficiencies and, possibly, other genetic diseases” of the blood, the editorialists conclude.
SOURCE: The Lancet, December 18/25, 2004.
Revision date: July 4, 2011
Last revised: by Jorge P. Ribeiro, MD