For decades, a Huntington’s disease diagnosis has been a death sentence. It shows up like a wrecking ball in midlife, disrupting motor function, cognition, and emotional stability. It grinds its victims down over 10 to 25 brutal years, with treatments only masking symptoms.
However, after years of work and numerous failed trials, researchers may have finally developed a viable treatment. In clinical trial results released on September 24, a gene therapy called AMT-130 became the first treatment actually to slow the progression of Huntington’s disease.
It doesn’t just manage the fallout. That’s a potentially massive game-changer for the around 40,000 Americans with Huntington’s disease.
The First Actual Treatment for Huntington’s Disease Might Finally Be Here
AMT-130 is a gene therapy. Scientists take a harmless virus, pack it with microRNA designed to silence the mutant HTT gene, which is responsible for producing the toxic protein behind the brain damage, and surgically inject it deep into two parts of the brain. It’s a one-and-done procedure. You get one dose, one surgery, and potentially years of slowed decline after that.
In the 17 patients given a high dose and tracked over three years, progression slowed by 75 percent compared to those on standard treatment. In some cases, levels of neurofilament light protein, which act as a biomarker of brain cell death, actually dropped.
Dr. Victor Sung, a leading researcher on the landmark trial, said that the data represent “some of the most compelling in all of neurodegenerative disease.”
It’s still early. The trial is small, the effects might fade, and access to it all could be limited due to cost and the complexity of the surgery. But this is nothing but tangible good news for a community of sufferers who have subsisted on hope for too long.
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