Has the world’s first gene therapy changed a three-year-old’s life?

WEB DESK: A groundbreaking medical milestone has given new hope to families affected by a rare genetic disorder, as a three-year-old boy from California became the first child in the world to receive an experimental gene therapy for Hunter syndrome, and has shown remarkable improvement a year later.

Oliver, diagnosed with MPS II (Hunter syndrome), suffered from a condition so severe it is often described as “childhood dementia.” The rare inherited disorder damages both the brain and body, and most children with its aggressive form do not survive beyond adolescence. Oliver’s older brother, Skyler, also lives with the disease, a discovery that the family describes as the most devastating moment of their lives.

According to a BBC report, doctors in Manchester began treating Oliver in late 2024 by extracting stem cells from his blood. Previously, only one treatment existed, costing nearly £300,000 per year, and crucially, it could not reach the brain, meaning cognitive decline continued despite therapy.

Oliver’s stem cells were transported to Great Ormond Street Hospital in London, where scientists inserted a healthy copy of the IDS gene. This corrected the enzyme deficiency at the root of the disease.

Months later, more than 120 million genetically modified stem cells were infused back into Oliver’s body through two small doses administered within an hour. The procedure itself lasted only minutes but marked a turning point in his life.

One year on, Oliver is developing like a typical child. Lead clinical investigator Professor Simon Jones said, “I’ve waited 20 years to see a child respond like this. Oliver’s improvement is extraordinary.”

Oliver is the first among five children selected globally for this pioneering trial, and the therapy’s early success is being hailed as a breakthrough offering new hope to families worldwide living with Hunter syndrome.