Doctors say Huntington's disease has been successfully treated for the first time, offering hope for those affected by the inherited fatal condition. The condition, which typically strikes in a person's 30s or 40s and progresses over about 20 years, causes severe neurological decline, per the BBC. In a recent clinical trial, a new gene therapy from UniQure slowed the disease's progression by an average of 75% three years after surgery. That means the functional decline expected over a single year could now take four years after treatment, potentially giving patients decades of improved quality of life, according to Sarah Tabrizi of University College London.
The experimental therapy involves a complex procedure that involves from 12 to 18 hours of surgery, in which doctors infuse a modified virus carrying a specially designed DNA sequence into two specific brain regions. Once inside, this genetic material prompts the brain cells to produce molecules that suppress the faulty huntingtin protein responsible for the disease. "For people living with Huntington's, an effective, one-time therapy ... could preserve years of quality relationships and gainful employment that would normally be lost to the disease," notes STAT.
Indeed, doctors reporting back after the small trial of 29 patients said that some participants who were expected to need wheelchairs are still walking, and one patient, previously retired due to the illness, has returned to work. The treatment appears to be safe, though some patients experienced temporary inflammation. The Independent notes that a single dose should last a person for life.
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While the results are promising, the therapy is expected to be expensive and available to only a limited number of patients initially, due to the complexity of the surgery. Regulatory approval is being sought, with a US launch targeted for late 2026. Researchers are also exploring whether treatment before symptoms appear could delay or prevent Huntington's entirely.
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