It's been a big month for gene therapy: first a breakthrough for leukaemia last week, now today scientists announced they've successfully treated kids with "bubble boy" disease.
We've heard this before — back in 2000 gene therapy successfully treated the same immune disease, but by 2007 five out of the 10 boys had developed leukaemia (ironically, the disease successfully treated in the study announced last week), and one died. This time, the researchers smartly waited NINE YEARS to declare success with 14 out of 16 boys. It's hard to argue with nearly a decade of good health.
The boys reportedly are in school like normal kids, which without treatment could have killed them. That's because they have an immune disorder called severe combined immunodeficiency, or SCID, a rare inherited disease that leaves them with basically no immune protection. (You know The Boy in the Plastic Bubble was true, right?).
Today's studies (there were two, both published in Science Translational Medicine) is exciting news both for boys (only boys inherit the gene that causes it, and many die in infancy) with the disease but also for gene therapy, which researchers have been trying to make work for 20 years. Jesse Gelsinger's death following a gene therapy clinical trial in 1999 was a huge setback, as was the development of leukaemia in the SCID boys after the 2000 clinical trials.
Scientists have modified the gene therapy to try to prevent patients from developing leukaemia, but the risk of developing the disease still exists — one boy in the latest study developed it. But parents are choosing gene therapy over the alternatives: a bone marrow transplant could cure SCID, but only one in five patients finds a match; with a partial match, one in three will die.
[Science Translational Medicine]