Italian researchers have successfully used treatments derived from the AIDS virus to treat children with two serious genetic diseases, it was announced last night.
Researchers say there is now evidence the treatment has been effective in helping six children and succeeded in halting the progress of disease.
The treatment involves "infecting" bone marrow cells from the children with a portion of an HIV virus carrying healthy forms of gene.
It has been used for a condition called Wiskott-Aldrich Syndrome, which causes immune system defects and bleeding risk and for a condition called metachromatic leukodystrophy, which affects the brain and nervous system.
The researchers said the choice of virus was the key to the success of the treatment. Other attempts at gene therapy have proved ineffective and in some cases have caused leukaemia.
The findings were reported in Science last night.
Luigi Naldini, who has led the research project at the San Raffaele Telethon Institute, Milan, said: "Three years after the start of the clinical trial the results obtained from the first six patients are very encouraging: the therapy is not only safe, but also effective and able to change the clinical history of these severe diseases.
"After 15 years of effort and our successes in the laboratory, but frustration as well, it’s really exciting to be able to give a concrete solution to the first patients."
Alessandro Aiuti, who has led the research on Wiskott-Aldrich syndrome, said: "Thanks to gene therapy, the children no longer have to face severe bleeding and infection. They can run, play and go to school."
Science 11 July 2013
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