So the team monitored a group of 14 SCID children who had been treated with gene therapy, and compared them to another group of 13 who had received the half-matched transplant. “To ensure that we are providing the best alternative therapy possible, we wanted to compare outcomes among infants treated with gene therapy and infants receiving partial matched transplants.” The study’s lead author, Fabien Touzot, explained the importance of comparing these two methods: That all changes today, as scientists at the Necker Children’s Hospital in Paris compare in the journal Blood, for the first time, half-matched transplants and gene therapy-to see which approach comes out on top. But are the second-tier contenders-gene therapy and half-matched transplant-both equally as effective? Until recently, no one had direct comparison. Experts still consider a fully matched stem cell transplant to be the gold standard of treatment for SCID.
#Bubble boy disease trial
Late last year, we wrote about a promising clinical trial from UCLA researcher (and CIRM Grantee) Donald Kohn, whose team effectively ‘cured’ SCID in 18 children with the help of gene therapy. But now, scientists are exploring a third, potentially advantageous option: gene therapy. Until recently, the next best option was a ‘half-match’ transplant-usually from a parent. But as you may have guessed, finding a match is extraordinarily rare. The only way to treat the disorder was with a fully matched stem cell transplant from a bone marrow donor, ideally from a sibling. This is the reality of SCID, also called “Bubble Baby” disease, a term coined in the 1970s when the only way to manage the disease was isolating the child in a super clean environment to avoid exposure to germs. He died in 1984 at 12 due to complications from a bone marrow transplant.
Born in 1971 with SCID, David Vetter lived in a sterile bubble to avoid outside germs that could kill him.
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