Medical breakthrough: Could this new treatment finally end one of the world’s deadliest diseases?
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For generations, families affected by Huntington’s disease have carried a painful burden.
This inherited disorder robs people of their movement, memory, and independence, often striking in the prime of life.
Now, for the very first time, doctors have achieved a medical milestone that slows the disease’s devastating impact.
The breakthrough offers a powerful sense of hope for tens of thousands of Americans and countless more around the globe.
If you’re not familiar with Huntington’s, you’re not alone. Often described as a cruel blend of dementia, Parkinson’s, and motor neurone disease, Huntington’s is a progressive brain disorder caused by a faulty gene.
This gene produces a mutant protein—aptly named “Huntingtin”—that slowly kills brain cells. The result? A relentless decline in physical, mental, and emotional abilities.
The numbers are sobering: about 41,000 Americans are currently living with Huntington’s, and every child of an affected parent faces a 50/50 chance of inheriting the gene.
Symptoms usually appear between ages 30 and 50, and until now, there’s been no way to stop the disease in its tracks.
That’s why the recent announcement from University College London (UCL) is nothing short of spectacular.
In a groundbreaking clinical trial, researchers managed to slow the progression of Huntington’s by a staggering 75%.
To put that in perspective: what would have been a year of decline now takes four years. For families living in the shadow of Huntington’s, that’s not just a statistic—it’s a lifeline.
Professor Sarah Tabrizi, director of the UCL Huntington’s Disease Centre, summed up the mood: “We never in our wildest dreams would have expected a 75% slowing of clinical progression.”
The science behind the breakthrough is as fascinating as it is complex.
The treatment uses gene therapy, delivered directly into the brain during a marathon 12- to 18-hour surgery. Here’s how it works:
The trial involved 29 brave patients, and three years later, the results are nothing short of remarkable.
Not only has the progression of the disease slowed, but some participants have even regained abilities they thought were lost forever.
One man, previously forced into early retirement, has returned to work. Others, who were expected to be in wheelchairs, are still walking.
Professor Ed Wild, a consultant neurologist at UCL, called it “the result we’ve been waiting for.”
Also read: Scientists say one vitamin supplement could help reduce skin cancer risk
For people like 30-year-old Jack May-Davis, this isn’t just a medical headline—it’s a new lease on life.
Jack inherited the Huntington’s gene from his father, Fred, who passed away in 2016 after years of decline.
Jack remembers the heartbreak of watching his dad’s health deteriorate, requiring round-the-clock care.
Now, after participating in the UCL trial, Jack is “overwhelmed” with hope. “It does allow me to think my life could be that much longer,” he shared.
For the first time, he can plan for a future that once seemed out of reach.
Also read: One small change could support your liver—and experts say it’s worth it
Of course, this therapy isn’t a magic bullet—at least not yet. The treatment is expected to be expensive and may not be suitable for everyone.
But the company behind the therapy, UniQure, is already preparing to seek accelerated approval from the US Food and Drug Administration (FDA) next year, with plans to expand to the UK and Europe soon after.
If approved, this could mark the beginning of a new era—not just for Huntington’s, but for other genetic brain diseases as well.
Gene therapy is a rapidly evolving field, and this success story could pave the way for similar breakthroughs in conditions like ALS, Alzheimer’s, and beyond.
Read next: New clue could help detect one of the deadliest cancers sooner, doctors say
Have you or your family been affected by Huntington’s disease? What does this breakthrough mean to you? Do you have questions about gene therapy or other advances in medicine?
This inherited disorder robs people of their movement, memory, and independence, often striking in the prime of life.
Now, for the very first time, doctors have achieved a medical milestone that slows the disease’s devastating impact.
The breakthrough offers a powerful sense of hope for tens of thousands of Americans and countless more around the globe.
If you’re not familiar with Huntington’s, you’re not alone. Often described as a cruel blend of dementia, Parkinson’s, and motor neurone disease, Huntington’s is a progressive brain disorder caused by a faulty gene.
This gene produces a mutant protein—aptly named “Huntingtin”—that slowly kills brain cells. The result? A relentless decline in physical, mental, and emotional abilities.
The numbers are sobering: about 41,000 Americans are currently living with Huntington’s, and every child of an affected parent faces a 50/50 chance of inheriting the gene.
Symptoms usually appear between ages 30 and 50, and until now, there’s been no way to stop the disease in its tracks.
That’s why the recent announcement from University College London (UCL) is nothing short of spectacular.
In a groundbreaking clinical trial, researchers managed to slow the progression of Huntington’s by a staggering 75%.
To put that in perspective: what would have been a year of decline now takes four years. For families living in the shadow of Huntington’s, that’s not just a statistic—it’s a lifeline.
Professor Sarah Tabrizi, director of the UCL Huntington’s Disease Centre, summed up the mood: “We never in our wildest dreams would have expected a 75% slowing of clinical progression.”
The science behind the breakthrough is as fascinating as it is complex.
The treatment uses gene therapy, delivered directly into the brain during a marathon 12- to 18-hour surgery. Here’s how it works:
- A modified virus is used as a delivery vehicle, carrying new genetic instructions into the brain.
- These instructions spread from cell to cell, teaching them to block the production of the toxic Huntingtin protein.
- The result? Fewer brain cells die, and the disease’s relentless march is dramatically slowed.
The trial involved 29 brave patients, and three years later, the results are nothing short of remarkable.
Not only has the progression of the disease slowed, but some participants have even regained abilities they thought were lost forever.
One man, previously forced into early retirement, has returned to work. Others, who were expected to be in wheelchairs, are still walking.
Professor Ed Wild, a consultant neurologist at UCL, called it “the result we’ve been waiting for.”
Also read: Scientists say one vitamin supplement could help reduce skin cancer risk
For people like 30-year-old Jack May-Davis, this isn’t just a medical headline—it’s a new lease on life.
Jack inherited the Huntington’s gene from his father, Fred, who passed away in 2016 after years of decline.
Jack remembers the heartbreak of watching his dad’s health deteriorate, requiring round-the-clock care.
Now, after participating in the UCL trial, Jack is “overwhelmed” with hope. “It does allow me to think my life could be that much longer,” he shared.
For the first time, he can plan for a future that once seemed out of reach.
Also read: One small change could support your liver—and experts say it’s worth it
Of course, this therapy isn’t a magic bullet—at least not yet. The treatment is expected to be expensive and may not be suitable for everyone.
But the company behind the therapy, UniQure, is already preparing to seek accelerated approval from the US Food and Drug Administration (FDA) next year, with plans to expand to the UK and Europe soon after.
If approved, this could mark the beginning of a new era—not just for Huntington’s, but for other genetic brain diseases as well.
Gene therapy is a rapidly evolving field, and this success story could pave the way for similar breakthroughs in conditions like ALS, Alzheimer’s, and beyond.
Read next: New clue could help detect one of the deadliest cancers sooner, doctors say
