Boy, 3, becomes first-ever person to receive groundbreaking gene therapy for hunter syndrome
A three-year-old boy has shocked doctors by becoming the first person ever to receive a groundbreaking gene therapy for Hunter syndrome.
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Hunter syndrome, a genetic disorder affecting around one in 100,000 boys, sees children slowly use their cognitive and physical abilities.
Oliver Chu was diagnosed with the devastating illness aged two and was expected to live into his teenage years.
But in a remarkable turn that has shocked the medical world, a new treatment developed in Manchester has seemingly reversed the condition, giving the American toddler the chance of a normal life.
Ricky Chu, Oliver’s father, said: “He’s like a completely different child. He’s running around everywhere, he won’t stop talking. The future for Ollie seems very bright and hopefully this means more kids will get the treatment.”
Young Oliver’s mum, Jingru, added: “Every time we talk about it I want to cry because it’s just so amazing.”
Prior to receiving the gene treatment, Oliver was unable to produce an enzyme crucial to living a normal life, which leaves him unable to break down complex sugars, causing organ damage, joint stiffness, hearing loss and breathing difficulties.
Mr Chu previously said: “When you find out about Hunter syndrome, the first thing the doctor tells you is, ‘Don’t go on the internet and look it up because you’ll find the worst cases and you’ll be very, very disheartened’.
“But, like anybody, you look it up and you’re like, ‘Oh my goodness, is this what’s going to happen to both my sons?’”
Oliver’s age was crucial to the treatment being effective, Professor Simon Jones, an expert at the Manchester Centre for Genomic Medicine, said.
“For this trial, we wanted to treat children really early, because we thought that would give them the best chance of a normal outcome,” Mr Jones told The Times.
Most children are diagnosed too late for the treatment to work, he explained, meaning several British families were turned away.
“It was really tragic,” he said. “There are lots of tragic stories that are part of this success.”
Prior to this breakthrough, the only treatment for Hunter syndrome was Elaprase, which costs each patient £300,000 per year.
And while this new treatment is still expensive, Mr Jones believes it could save hundreds of lives in the long term.