Gene Therapy Slows Huntington's Disease in First Trial

For families watching loved ones slowly lose control of their movements, thoughts, and emotions to Huntington's disease, decades of "no cure available" have felt like a life sentence. Now, researchers at University College London have finally broken through with a treatment that actually slows the disease down.

The gene therapy, called AMT-130, targets the root cause of Huntington's. It reduces production of the toxic mutant protein that kills brain cells in patients carrying the faulty gene.

In a three-year trial with 29 patients, the results exceeded expectations. People receiving the therapy showed slower cognitive decline, particularly in processing speed and reading ability. Even more exciting, their levels of neurofilament light, a marker showing active brain cell damage, dropped below baseline levels.

That last finding matters enormously. It suggests the therapy isn't just masking symptoms but actively protecting the brain from destruction.

Huntington's strikes cruelly early, typically appearing when people are between 30 and 50 years old, still working and raising families. Patients usually live 15 to 20 years after diagnosis, gradually losing the ability to move, think clearly, and control their emotions.

Scientists discovered the genetic cause back in 1993, a repeating DNA sequence in the Huntingtin gene. Healthy people have fewer than 35 repeats of the letters C-A-G. More than 39 repeats guarantees someone will develop the disease. At the time, everyone thought a cure would come quickly. It didn't.

But researchers kept studying people decades before symptoms appeared. They found brain changes starting 15 to 25 years before the first tremor or stumble, particularly in the striatum, the brain region controlling movement.

A new study from UCL and Cambridge followed 64 people with the Huntington's gene expansion roughly 24 years before expected symptoms. Even at this early stage, researchers spotted mild disruptions in cognitive flexibility and changes in brain connectivity. When they checked back 4.5 years later, the changes had become clearer.

Why This Inspires

These studies paint a picture of possibility that didn't exist five years ago. Doctors can now spot the disease decades before symptoms appear and may soon have treatments to offer during that window.

For someone who just learned they carry the Huntington's gene, this changes everything. Instead of watching the calendar count down to inevitable decline, they might receive treatment that preserves their ability to think, move, and live fully for years longer than previously possible.

The gene therapy still needs full peer review and larger trials. But after more than a century since Huntington's disease was first described, families finally have something researchers couldn't offer before: real, measurable hope.