Breakthrough Treatment

A new gene therapy has become the first treatment to successfully slow the progression of Huntington’s disease.

Context

Huntington’s disease is a devastating inherited brain disorder that typically strikes people in their 30s or 40s. The condition causes brain cells to die progressively, leading to uncontrolled movements, cognitive decline, dementia, and eventually death within about two decades. Those who have a parent with Huntington’s have a 50% chance of inheriting the disease, which affects between 6,000 and 10,000 people in the UK and about 40,000 Americans with symptoms.

Treatment

Researchers at University College London released early results this week from an ongoing Huntington’s treatment study. The experimental therapy slowed disease progression by 75% in patients after three years, meaning the decline that would normally happen in one year was stretched over four years instead.

The treatment, developed by Dutch biotech company uniQure, stops the production of toxic proteins that damage brain cells using a harmless virus to deliver genetic material into neurons. Once inside, this material instructs the cells to block production of the harmful huntingtin protein that causes the disease. 

Delivery Process

The treatment requires a complex surgical procedure lasting 12 to 18 hours, during which doctors use real-time brain scans to guide a thin catheter deep into two specific brain regions.

The therapy is delivered as a single injection that appears to permanently reduce levels of the toxic protein. While the procedure was generally well-tolerated, some patients experienced headaches and confusion that either resolved on their own or required steroid treatment.

Path to Availability

UniQure announced plans to submit the therapy for regulatory approval in the US in early 2026, with a potential launch before 2027 if approved. The treatment will likely be expensive due to the complex surgery required.

Professor Sarah Tabrizi, who led the trial at University College London, said she was “really overjoyed” and called the development “absolutely huge,” noting that many more people might now seek genetic testing since an actual treatment exists, rather than just symptom management.