Preliminary but promising gene therapy shows 75% reduction in Huntington's disease progression

Scientists have reported the first therapy to slow the progression of Huntington's disease, based on a preliminary trial from University College London (UCL). The experimental one-time gene therapy, called AMT-130, showed a 75% reduction in disease progression over three years for patients receiving a high dose compared to those on standard care. While these results are promising, the treatment is still years away from potential FDA approval, with the earliest application expected in 2026.

The therapy works by using a harmless virus to deliver genetic instructions to the brain, which turn off the harmful protein responsible for damaging brain cells. Unlike existing treatments that only manage symptoms, AMT-130 is designed as a lifelong, one-time intervention. In the study involving 29 patients, with 12 receiving a high dose, success was measured through brain scans and spinal fluid tests showing significant improvement in reducing brain cell damage.

Huntington's disease affects approximately 100,000 Americans and is caused by a faulty gene that leads to toxic proteins attacking brain cells, typically appearing in mid-adulthood and progressing over 15-20 years with symptoms like movement issues, memory loss, and mood changes. Experts involved in the trial, such as UCL professors Ed Wild and Sarah Tabrizi, expressed excitement, noting that the therapy could help patients preserve daily function and even return to work, though larger studies are needed to confirm these early findings.

Despite the encouraging outcomes, researchers caution that the treatment requires complex brain surgery to deliver the therapy directly to affected areas, though patients tolerated it well with manageable side effects. If approved, AMT-130 would be the first treatment to slow the disease itself rather than just alleviate symptoms. The findings have not yet been published in a medical journal or peer-reviewed but will be presented at an upcoming conference, highlighting the need for further testing and review for safety and effectiveness.