Huntington's disease successfully treated for the first time

For the first time, doctors say they have successfully treated Huntington’s disease — one of the cruellest and most devastating genetic conditions. The news, first reported by BBC, has been described as “spectacular” by researchers who witnessed patients regain quality of life once thought impossible.

What is Huntington’s disease?

Huntington’s disease is a hereditary disorder that destroys brain cells and combines features of dementia, Parkinson’s and motor neurone disease. It usually strikes in a person’s 30s or 40s and is considered fatal within two decades. A faulty gene, known as the huntingtin gene, transforms a vital brain protein into a killer of neurons. If one parent has the defective gene, their children face a 50% chance of inheriting it.




The disease manifests across three fronts: movement disorders(chorea), cognitive decline, and mental health impact.

A treatment once unthinkable

The new therapy is a type of gene therapy delivered during 12 to 18 hours of intricate neurosurgery. A safe, modified virus carries a custom DNA sequence into the brain, guided by MRI to two key regions: the caudate nucleus and the putamen. Once inside, neurons begin producing microRNA fragments designed to silence the mutant huntingtin protein at its source.



The therapy aims to permanently lower toxic protein levels in a single dose. Data released by the biotech firm uniQure showed that three years after surgery, patients experienced a 75% slowing of disease progression. On average, a year’s decline would now take four years, offering the potential for decades of good-quality life.



“This is the result we've been waiting for,” said Prof Ed Wild, consultant neurologist at University College London Hospitals. “There was every chance that we would never see a result like this, so to be living in a world where we know this is not only possible, but the actual magnitude of the effect is breathtaking, it's very difficult to fully encapsulate the emotion.”

The human impact

The trial, which involved 29 patients, has already transformed lives. One patient who had been forced into medical retirement has returned to work. Others who were expected to be in wheelchairs are still walking.

Jack May-Davis, 30, knows the weight of Huntington’s all too well. His father, Fred, first showed symptoms in his late 30s, eventually requiring round-the-clock care before his death at 54 in 2016. Jack inherited the faulty gene and had long assumed he was destined for the same fate. But today, he is hopeful.

Looking ahead

While the therapy appears safe, some patients did experience inflammation caused by the viral delivery, leading to headaches and confusion treatable with steroids. Despite the challenges, Prof Wild believes the treatment “should last for life” since brain cells are not naturally replaced.



Gene therapies are notoriously expensive; in the UK, the NHS funds one for haemophilia B that costs £2.6 million per patient, while in the USA it is Hemgenix, a gene therapy, costing $3.5 million per dose in the USA.