'Bubble boy' gene therapy works long-term in major study
A major study just found that a single gene therapy can cure 95% of children with ADA-SCID, also known as "bubble boy" disease—a rare, life-threatening immune disorder.
The treatment uses each child's own stem cells, genetically fixed to restore their immune system for good, so no more need for donor transplants or lifelong enzyme treatments.
All kids survived and most needed no further treatment
Between 2012 and 2019, 62 kids in the US and UK got mild chemo and then their gene-modified stem cells.
After about 7.5 years, every child survived and almost all needed no further treatment.
Their immune systems bounced back, handled vaccines well, and no serious side effects showed up.
Frozen stem cells make the therapy easier to access
Over half the kids received frozen stem cells, which means the cells can be collected and processed locally—making the therapy easier to access, even if you're not near a big hospital.
All children were followed for a median of 7.5 years after treatment, with no mention of follow-up beyond a decade.
FDA approval could come in the next couple of years
With these results, FDA approval for this gene therapy could come in the next 2-3 years (by 2027 or 2028), opening up a real cure for kids and families dealing with ADA-SCID worldwide.