A three-year-old boy with the devastating inherited condition Hunter syndrome (MPSII) has achieved “astounding” progress following a world-first gene therapy administered in Manchester, UK.
Oliver Chu, from California, became the first patient globally to receive the pioneering treatment designed to halt the progressive damage caused by the disease, often described as a form of childhood dementia.
The Ground-Breaking Treatment
- The Problem: Hunter syndrome prevents the body from producing a crucial enzyme (iduronate-2-sulfatase or IDS), leading to the accumulation of sugar molecules that progressively damage the brain and body. Patients often die before age 20.
- The Solution: Medical staff at Royal Manchester Children’s Hospital genetically modified Oliver’s own stem cells in a lab at GOSH, inserting the missing IDS gene. Crucially, the gene was modified to ensure the resulting enzyme could cross the blood-brain barrier—a major limitation of previous treatments.
- The Result: A year after the one-off infusion of 125 million gene-modified cells, Oliver is now producing his own essential enzymes and appears to be developing normally, with dramatically improved speech, mobility, and cognitive development.
Hopes for the Future
“It’s just so amazing,” said Oliver’s mother, Jingru, noting her son is “so different” and “talking a ton.” Prof. Simon Jones, co-leading the trial, called Oliver’s progress “so exciting,” noting that the boy is now making hundreds of times the normal amount of the missing enzyme.
The success offers enormous hope for other children, including Oliver’s older brother, Skyler, who also has Hunter syndrome. Five boys from around the world have been enrolled in the trial, which almost failed due to lack of funding before being saved by a £2.5m commitment from the charity LifeArc.
