US based researchers announced that they have cured 10 children of severe combined immunodeficiency disorder, known as the Bubble Boy disease, using an altered version of HIV
Doctors at St Jude Children’s Research Hospital, USA, claim that they have successfully cured the ‘bubble-boy’ disease in 10 infants using an altered version of HIV. The disease, termed as X-linked severe combined immunodeficiency (SCID-X1), is a rare a genetic disorder that renders the patient’s immune system completely defenceless.
These new findings are a step forward in the current gene replacement therapy, the earlier versions of which has been known to be less efficient procedure that puts the patients at a high risk of leukaemia. St Jude doctors claim that with this latest development, they have significantly reduced the risk for the patient to develop leukaemia by planting “insulators” around the replaced genes, rendering the treatment unable to accidentally trigger the surrounding genes and cause cancer spread.
Out of 10 children treated, nine were found to have developed non-defective immune cells after three months. The child which did not show this progress was given a second treatment after completing one year and is reported to have developed a healthy immune system as well.
It can take up to three to four months for treated patients to develop fully functional immune systems, after which the patients can leave isolation.
Though the doctors are confident of the new treatment’s ability to cure SCID-X1 with the new procedure, experts believe that its durability is yet to be tested by time.
Nevertheless, the procedure is “a significant step forward in the development of gene therapy and specifically for these diseases,” according to Dr Alain Fischer, Professor, College of France, who has been involved in the development of the gene therapy for over two decades.
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