HUNTINGTON’S disease has been successfully treated for the first time in a “world-changing” trial, doctors say.
The incurable illness causes decades of nerve damage that destroy the brain and cripple the body.
The new treatment involves a brain operation lasting between 12 and 20 hours (stock image)[/caption]
A gene therapy trial on 29 patients now claims to have slowed the disease by 75 per cent, meaning one year’s worth of decline could be spread over four years.
Experts said they have never seen anything like this and it could offer hope to thousands of people and families afflicted by Huntington’s in the UK.
Around 7,000 people are estimated to have it and it is inherited through families.
Results were announced by pharmaceutical company uniQure and University College London’s Huntington’s Disease Centre.
The treatment AMT-130 involves a one-off 12 to 20-hour brain surgery to inject a special type of DNA to block the genes causing the disease to worsen.
It is not a cure but appears to slow the illness, which was previously impossible meaning patients often died painfully in middle age.
American country singer Woody Guthrie, writer of This Land is Your Land, died from Huntington’s disease aged 55 in 1967.
Singer Woody Guthrie died of Huntington’s disease in 1967[/caption]
The Sound of Music star, Dame Julie Andrews, has campaigned for research as a board member of the Huntington’s Disease Foundation.
Study leader at UCL, Professor Sarah Tabrizi, said: “This is going to keep people in work longer, functioning longer, delaying symptoms of disability – this is truly a game-changing result.
“It is huge. I’ve never seen anything that slows functional decline.”
Professor Ed Wild, also from UCL, called it “truly world-changing stuff”.
Dr Walid Abi-Saab, chief medical officer of uniQure, added: “We are incredibly excited about these results and what they may represent for individuals and families affected by Huntington’s disease.”
What is Huntington’s disease?
HUNTINGTON’S disease is rare, affecting about eight in every 100,000 people in the UK.
It stops parts of the brain working properly over time, which can impact movement and cognition.
Symptoms usually start between the ages of 30 and 50, but it can begin much earlier or later.
People with Huntington’s might notice:
- Difficulty concentrating and memory lapses
- Depression
- Stumbling and clumsiness
- Involuntary jerking or fidgety movements of the limbs and body
- Mood swings and personality changes (e.g. irritability or aggressive behaviour)
- Problems swallowing, speaking and breathing
- Difficulty moving
Signs can be subtle at first, but full-time nursing care is needed in the later stages.
Huntington’s is caused by a faulty gene that results in parts of the brain becoming gradually damaged over time.
It is inherited, so you are usually only at risk of developing it if one of your parents has or had it.
If a parent has the Huntington’s gene, there’s a 50 per cent chance each of their children will develop the condition and pass it on to their kids.
There is currently no cure, but medication and therapy can help reduce some of the symptoms.
Source: NHS
