Hope for people with Huntington's disease

Hope for Huntington's patients - 27th October 2025

For the first time, Huntington's disease has been successfully treated. A cruel disease, it impacts the body's whole nervous system both physically and mentally. Caused by an error in our genetic material, the huntingtin gene (HTT) runs through families. The child of a parent with Huntington's disease has a one in two chance of developing it.

The HTT gene produces an important protein needed by nerve cells in the brain. However, a gene mutation turns the huntingtin protein into a neuron killer, shortening sufferers' life expectancy.

The genetic basis of Huntington's was first recognised in 1993, yet until now there has been no cure or treatment available. However, a company specialising in gene therapy called uniQure, together with scientists from University College London (UCL) have achieved very positive results.

The treatment process involved 29 individuals and required a type of gene therapy necessitating 12 to 18 hours of delicate brain surgery. The final results shocked even those directly involved. Professor Sarah Tabrizi, director of the Huntington's Disease Centre at UCL said, "We never in our wildest dreams would have expected a 75 percent slowing of clinical progression."

It means the decline normally expected in one year after treatment would take four years. And that could give patients decades of "good quality life". In fact, Professor Ed Wild, neurologist at UCL Hospitals, believes the effects could last for life.

The treatment is costly and won't be widely available due to the highly complex surgery. Professor Tabrizi, however, assumes that it will open doors to other therapies.