Hope for people with Huntington's disease

Hope for Huntington's patients - 27th October 2025

For the first time, people with Huntington's disease have been given real hope. Huntington's is a serious disease of the brain and nervous system, which brings about an early death.

It's caused by an error in our DNA, in the 'huntingtin' gene. This gene normally produces proteins which are needed by the brain. However, with a change or 'mutation' in the gene, it actually kills nerve cells.

Huntington's is a disease which runs in families. So a child with a parent who has this disease has a 50 percent chance of developing it.

Information about the genetic problems was first identified in 1993. However, until now research hasn't produced a cure. A programme by a gene therapy organisation, uniQure, and University College London (UCL) has now had positive results.

The new treatment, a kind of gene therapy, was given to 29 people. It took 12 to 18 hours of careful brain surgery but the results were surprising, even for the team. The treatment slowed the development of the disease by 75 percent. Professor Sarah Tabrizi, from UCL's Huntington's Disease Centre said it could give patients years of "good quality life".

One brain expert from UCL, Professor Ed Wild, is even more hopeful. He thinks there's a possibility the treatment will last for life.

The treatment isn't cheap and it's extremely complicated. Yet Professor Tabrizi believes this is just the beginning.