Name: Boy with rare condition amazes doctors after world-first gene therapy | BBC News
Uploaded: 2025-11-24T16:55:26.000Z
Duration: 7 min 28 s
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A three year old boy from California with a rare debilitating condition has become the first person in the world to be treated with a gene therapy developed in Manchester.

Ollie Chu has Hunter syndrome, which causes progressive damage throughout the body.

Our medical editor, Fergus Walsh has his story.

The future for Ollie seems very bright and hopefully this means more kids will get the treatment and, you know, the future will be bright for them.

How science is taking on a brutal inherited disease.

We're so happy, so pleased with his progress.

His results are amazing and what the new treatment means to those affected.

It's really amazing how they this treatment had achieved and you know, how he is so different now than before.

Oliver has Hunter syndrome, also called MPS2.

A faulty gene means he can't produce a crucial enzyme.

This means chemicals build up in cells, causing progressive damage throughout the body.

Usually only boys are affected by this rare inherited condition.

Boys, when they're born, usually look really well and healthy.

But gradually, over time, those chemicals build up and they gradually cause each part of our body, our bones, joints, brain, our airways to become more and more diseased.

Usually wheelchairs are required by the end of the first decade and the average survival was in the teenage years.

Oliver has flown with his dad from California to Royal Manchester Children's Hospital for the start of a groundbreaking medical trial.

You know, at the end of the day, we just want the kids to be, you know, healthy and have a more normal lifestyle than, you know, than they have right now.

Oliver is having his blood filtered to remove stem cells.

These special cells are going to be tweaked, genetically altered in the lab.

It's taken just over three hours to collect them, but this is just the first stage.

Oliver's cells are transferred to labs at London's Great Or Hospital.

Scientists use a harmless virus to insert a working copy of the faulty gene into each of Ollie's stem cells.

The gene gives instructions to the cells to start producing the missing enzyme, which should get to all parts of the body, including the brain, with the aim of halting further physical and, and

It's February and Oliver is back in Manchester, this time with his mum.

His genetically altered stem cells are here too.

The cells are slowly brought to body temperature.

Oliver is the first child in the world to be given this one off treatment to combat Hunter Syndrome.

Researchers at the University of Manchester and clinicians have spent 15 years developing the gene therapy.

So having an effective treatment that deals with all aspects of Hunter syndrome would be utterly life transforming for families.

It would mean moving from a constant life of medical interventions and slow decline and an early death to a life where there's hope.

Three months later, the entire family is over from California, including Oliver's brother Skylar, who's 5.

He also has Hunter syndrome, but was too old for this trial.

Skylar receives a weekly infusion of enzymes.

This helps prevent damage to the body, but they don't get to the brain.

By contrast, Oliver has been able to come off all treatment because the gene therapy is working.

I want to pinch myself every time I tell people, you know, Oliver is making his own enzymes.

Every time we talk about it, I wanted to cry because it's just so amazing.

My wish upon the star is for Skylar to be able to get the same treatment.

As devastating as the news was that Both kids had MPS2 hunter syndrome.

With this gene therapy, it feels like Oliver got a reset in his life and gets a start, you know, with almost a fresh slate, you know, and

that's why I want the same thing for Skylight.

The trial is funded by a medical charity, LifeArc.

Unusually, there's no pharmaceutical company involved.

Doctors remain cautious, but are thrilled.

The ambition is it could be a permanent fix for Hunter Syndrome.

I've been waiting 20 years for this, to see a boy like Ollie doing as well as he has, and

His spirit, his speech has gotten exponentially better, his mobility has gotten better, his agility, his cognitive, everything from his learning curve hasn't just gone up, but

So no more medication, no more infusions.

As close to a normal life as we can give him at this point.

Yeah, that's all we dream for for both our kids, honest.

Ollie is one of five boys who are part of this world first gene therapy trial.

if successful, it would transform their life chances and give hope to other families affected by this rare and often devastating condition.

Gene therapy is making huge strides in tackling rare diseases, so the outlook has never been so promising.

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Boy with rare condition amazes doctors after world-first gene therapy | BBC News

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