Breakthrough Gene Therapy Brings Hope to Children with Devastating Inherited Disorder
A groundbreaking gene therapy has offered a glimmer of hope for children like three-year-old Oliver Chu, who was the first boy to receive the treatment. Born with Hunter syndrome, a devastating inherited disorder that causes complex sugar molecules to accumulate in organs and tissues, Oliver's condition typically leads to joint stiffness, hearing loss, heart problems, and cognitive decline.
The only licensed treatment for Hunter syndrome is Elaprase, a weekly infusion that replaces the missing enzyme, costing £375,000 per patient. While it improves movement and organ problems, it doesn't reach the brain, meaning it cannot prevent cognitive decline. Oliver's parents are cautiously optimistic about the new therapy after seeing positive results in their son.
The treatment works by collecting stem cells from Oliver's blood, replacing his faulty gene with a working copy, and then re-infusing them back into his bloodstream. The corrected stem cells churn out high levels of the enzyme, which also reaches his brain. Since receiving the therapy, Oliver no longer needs weekly Elaprase infusions, an encouraging sign that the treatment is taking hold.
Doctors are cautiously optimistic about the progress, but it's too early to call the therapy a success. Prof Simon Jones, consultant in paediatric inherited metabolic disease at Manchester Centre for Genomic Medicine (MCGM), notes that while Oliver's results are promising, it's only been nine months since he received the treatment, and four more boys on the trial still need to be evaluated.
The gene therapy is being developed to treat other genetic disorders that impair vital enzymes, such as Hurler syndrome and Sanfilippo syndrome. For now, families like Oliver's hope that this breakthrough may also offer a lifeline for their children with these conditions.
A groundbreaking gene therapy has offered a glimmer of hope for children like three-year-old Oliver Chu, who was the first boy to receive the treatment. Born with Hunter syndrome, a devastating inherited disorder that causes complex sugar molecules to accumulate in organs and tissues, Oliver's condition typically leads to joint stiffness, hearing loss, heart problems, and cognitive decline.
The only licensed treatment for Hunter syndrome is Elaprase, a weekly infusion that replaces the missing enzyme, costing £375,000 per patient. While it improves movement and organ problems, it doesn't reach the brain, meaning it cannot prevent cognitive decline. Oliver's parents are cautiously optimistic about the new therapy after seeing positive results in their son.
The treatment works by collecting stem cells from Oliver's blood, replacing his faulty gene with a working copy, and then re-infusing them back into his bloodstream. The corrected stem cells churn out high levels of the enzyme, which also reaches his brain. Since receiving the therapy, Oliver no longer needs weekly Elaprase infusions, an encouraging sign that the treatment is taking hold.
Doctors are cautiously optimistic about the progress, but it's too early to call the therapy a success. Prof Simon Jones, consultant in paediatric inherited metabolic disease at Manchester Centre for Genomic Medicine (MCGM), notes that while Oliver's results are promising, it's only been nine months since he received the treatment, and four more boys on the trial still need to be evaluated.
The gene therapy is being developed to treat other genetic disorders that impair vital enzymes, such as Hurler syndrome and Sanfilippo syndrome. For now, families like Oliver's hope that this breakthrough may also offer a lifeline for their children with these conditions.
it's like oliver chu got a second chance at life! they took stem cells from his blood, fixed the faulty gene, and now he can walk & play w/o elaprase infusions 
but yea, its still early days & only 9 months in so we gotta see more progress before i start shouting from the rooftops 
but this is def some hope for kids like oliver who got stuck w/ hunter syndrome 
can't wait to hear more about dis gene therapy tho! 
can you imagine what it's like to have to spend £375k on treatment just to slow down your child's decline? 
elaprase is like a weekly lifesaver, but the good news about this new gene therapy is that it might become a game-changer for kids like oliver. if it works out, families won't have to break the bank every week and their little ones can actually get some progress in those brain cells 
the fact that it only takes 9 months to see results is amazing, and if they keep showing positive signs, we might just have a treatment that's not just life-prolonging but also helps with quality of life 
his story is so inspiring but also heartbreaking the thought of all those kids struggling with Hunter syndrome and not having any real treatment options is just devastating 
it's amazing that scientists are working on this gene therapy tho 
it's still early days but I'm keeping my fingers crossed for Oliver and all the other kids who might be able to benefit from this breakthrough 
over this gene therapy news!!! it's so cool to think that kids like oliver chu are finally getting a chance at a normal life without having to deal with hunter syndrome 
375k might seem like a lot, but can you imagine the peace of mind knowing your kiddo is going to live a longer, healthier life? 
and it's not just about oliver, this breakthrough could potentially change lives for so many more kids with similar conditions 
. The idea of a gene therapy that can replace faulty genes and produce an enzyme is just mind-blowing 
.
!! The thought of a treatment that can actually target the brain and prevent cognitive decline is just...wow. I mean, we've been talking about gene therapy for years, but to see it work in real life like this? It's a game-changer. And the fact that it's not just Oliver who's benefiting from this treatment, but also other kids with Hurler syndrome and Sanfilippo syndrome 
... I mean, £375,000 per patient? That's like, a small fortune! What's really going on with this gene therapy? Are they making a profit off our kids' suffering? 
And what about the trial results? Only been nine months? That's not enough time to know for sure if it's working. I'm not buying it... there's gotta be more to this story 
, but for Oliver and his family, it's a huge deal. Three years old and already seeing progress? That's amazing 
.
More research needed, I guess 
. Still, it's awesome to see progress on treatments like this 
! And Oliver's just a tiny beacon of hope for all those families out there struggling with these conditions 
. Fingers crossed it works out! 
. And £375,000 per patient? That's just outrageous! They're trying to make a profit off these poor kids' lives. Mark my words, there's more to this story than meets the eye 
.
. The fact that scientists have developed a treatment that can target the root cause of these diseases is truly remarkable 
They've found a way 2 help kids w/ Hunter syndrome & other rare disorders 
. I'm totes here 4 Oliver & his fam! 
. Four more boys need to be evaluated, so let's keep our fingers crossed for them too! Maybe one day we'll have treatments that can fix these gene issues before they even happen? That would be a game-changer