A single-dose gene therapy has demonstrated remarkable results in slowing the advancement of Huntington’s disease during a preliminary trial, according to a press release from University College London (UCL). Researchers reported that the experimental treatment, AMT-130, slowed disease progression by 75% over a three-year period in patients who received a high dose, compared with those who received standard treatment. Although the findings are encouraging, they are still preliminary, and the treatment may remain several years away from potential FDA approval, with the earliest submission anticipated in 2026.
Huntington’s disease impacts about 100,000 people in the United States and is caused by a defective gene that produces toxic proteins, leading to the destruction of brain cells. According to the press release, children of affected individuals have a 50% chance of inheriting the disorder. Symptoms typically begin in middle adulthood and include difficulties with memory, movement, and mood, with the disease usually worsening over 15 to 20 years.
Researchers explained that the new therapy uses a harmless virus to deliver targeted genetic instructions to the brain. These instructions work to shut down the damaging protein responsible for harming brain cells. Unlike current treatments that only address symptoms, AMT-130 is designed to be a lifelong, single-dose therapy.
The trial involved 29 participants, 12 of whom received a high dose of the therapy over three years. Scientists evaluated the treatment’s success using brain imaging and tests of spinal fluid that look for markers of brain cell damage — both of which showed notable improvement. Although the early findings are encouraging, experts emphasize the need for larger studies. The therapy must be delivered through complex brain surgery, but researchers noted that patients generally handled the procedure well and experienced manageable side effects.
If eventually approved, AMT-130 would be the first treatment to actually slow the progression of Huntington’s disease rather than simply treat its symptoms. Still, gaining FDA approval typically requires several more years of testing and review to confirm safety and effectiveness. The results have not yet been published in a peer-reviewed medical journal or evaluated by independent experts, but they are expected to be presented at a major medical conference next month.