Hope for people with Huntington's disease

Huntington's sufferers dare to hope - 27th October 2025

In a world first, Huntington's disease, the degenerative neurological disorder, has been treated successfully.

Symptoms of the disease resemble a combination of dementia, Parkinson's and motor neurone disease. It's a progressive illness brought about by an error in our DNA, specifically in the huntingtin gene (HTT). This gene is responsible for producing a vital protein required by brain neurons, but a gene mutation converts this protein into a neuron killer. And as the disease is hereditary, a child whose parent has Huntington's also has a 50 percent likelihood of inheriting it.

Although 1993 saw the analysis of the genetic information which led to diagnostic improvements, research had failed to provide a cure or to slow the disease's merciless progress. But a global trial sponsored by gene therapy firm uniQure and working in conjunction with scientists from University College London (UCL) has yielded outstanding results.

A total of 29 people agreed to participate in the clinical trial, which exploited a type of gene therapy administered during 12 to18 hours of meticulous brain surgery. The treatment's outcome has stupefied everyone, including the team themselves. Professor Sarah Tabrizi, director of UCL's Huntington's Disease Centre stated, "We never in our wildest dreams would have expected a 75 percent slowing of clinical progression." It means that the decline typically anticipated over just one year would take four years post treatment. This would allow "good quality life" to Huntington's sufferers.

Huntington's disease symptoms mainly emerge in a person's 30s or 40s and it's normally fatal within two decades.

Professor Ed Wild, consultant neurologist at the National Hospital for Neurology and Neurosurgery at UCL Hospitals, predicts the effects should last indefinitely, since brain cells aren't replaced as rapidly as other cells.

The high costs involved preclude wide-scale access to the treatment. However, Professor Tabrizi's convinced that this colossal achievement will open the gates for more therapies, enabling more people to procure life-saving treatment.