Doctors in the UK have developed a pioneering gene therapy, AMT-130, that slowed Huntington’s disease progression by 75%, marking the first effective treatment for the devastating genetic disorder.
Huntington’s disease treatment found
Doctors in the UK have successfully slowed the progression of Huntington’s disease for the first time, using a pioneering gene therapy called AMT-130. Results from a clinical trial show that the therapy reduced disease progression by 75% over 36 months, marking a turning point in the fight against one of the world’s cruellest genetic disorders.
Professor Sarah Tabrizi, director of the Huntington’s Disease Centre at University College London (UCL), who led the trial, described the findings as “absolutely huge” and said:
“We now have a treatment for one of the world’s more terrible diseases. This is absolutely huge. I’m really overjoyed.”
What is Huntington’s Disease?
Huntington’s disease is an inherited brain disorder caused by a mutation in the huntingtin gene. Each child of an affected parent has a 50% chance of inheriting it.
- It damages nerve cells in the brain, leading to a severe decline in movement, cognition, and mental health.
- Symptoms usually emerge in a person’s 30s or 40s and worsen steadily over 10–20 years.
- Signs include uncontrolled movements, memory decline, depression, anxiety, and obsessive behaviours.
- Because many people discover they carry the gene only after having children, the disease often devastates entire families across generations.
Huntington’s has long been called the “cruellest disease”, as it combines features of dementia, Parkinson’s, and motor neurone disease, with no existing cure to slow or stop its course.
How does the new therapy work?
The therapy, AMT-130, is a one-time gene treatment delivered during a 12–18-hour brain surgery.
- Doctors infuse a modified virus carrying a DNA sequence directly into two brain regions (the caudate nucleus and putamen).
- Once inside, the DNA switches off production of the toxic huntingtin protein, the root cause of the disease.
- A single dose is expected to last a lifetime, though the procedure is complex and likely to be expensive.
- In the trial, 29 patients in the UK and the US received treatment. Those given a high dose showed a dramatic slowing of progression, losing function four times slower than untreated patients.
Professor Ed Wild, principal investigator from UCL, said:
“The results change everything. On the basis of these results it seems likely AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff.”
Some patients have even regained independence: one who was medically retired has returned to work, while others remain mobile despite earlier predictions of wheelchair use.
What happens next?
The trial was conducted in partnership with uniQure, a biotech company based in the Netherlands and the US. The firm plans to submit data to the US Food and Drug Administration (FDA) in early 2026, paving the way for potential approval later that year.
Dr Walid Abi-Saab, chief medical officer of uniQure, said: “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease.”
If approved, it would become the first therapy to slow disease progression, offering patients not just more years of life but years with significantly better quality.
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FAQs on Huntington’s disease breakthrough
- What is Huntington’s disease? Huntington’s is a genetic brain disorder passed down through families. It develops when a single defective gene is inherited, and over time it causes steady damage to the brain’s nerve cells. People living with the condition often face difficulties with movement, memory, and mood. Once symptoms start, life expectancy is usually between 15 and 25 years
- How does the new treatment work? Doctors are testing a therapy called AMT-130. The treatment uses a modified virus, not to cause illness, but to carry new genetic material directly into the brain. The goal is to “switch off” the faulty huntingtin gene so the body produces less of the harmful protein that kills brain cells
- How effective is AMT-130?
Early trial results are promising. Over three years, patients who received the therapy experienced a 75% slowdown in the disease compared to those untreated. Some people on higher doses were able to keep their independence longer, and a few even returned to jobs they had given up.
- When might it be available? The company behind the treatment, uniQure, plans to ask the U.S. Food and Drug Administration for approval in 2026. If regulators agree, AMT-130 could become the first licensed drug to slow the disease’s progression, possibly reaching patients within the next couple of years.
NOTE – This article was originally published in Money Control and can be viewed here
