Huntington's Hopes

After years of heartbreak, a new treatment slows this devastating disease

- -Allison Parshall

This MRI shows Huntington's signature brain atrophy, which a new treatment may slow.

EVERY WEEK neurologist Victor Sung sees people with Huntington's disease, a rare and deadly neurodegenerative disorder, at his clinic at the University of Alabama at Birmingham. But a Wednesday last September was a day unlike any other. "I cried with every single patient," Sung says. The results of crucial phase 1/2 clinical trials had finally been released: an experimental gene therapy drug was the first treatment that appeared to slow the progression of Huntington's.

The treatment, known as AMT-130, is delivered deep into the brain during a surgery that lasts more than eight hours. The trials were small, with the three-year follow-up results including just 24 participants who received either a high or a low dose of treatment.

These results showed that a high dose reduced the rate of disease progression by 75 percent compared with rates in an external control group, according to the new therapy's developer, uniQure, which posted the results ahead of their review by the Food and Drug Administration. The company hopes to receive accelerated approval from the FDA, which, according to a uniQure spokesperson, could allow the drug to be approved by the end of 2026 without the need for phase 3 trials.

Sung's patients probably won't receive the drug anytime soon. But this early success has given the Huntington's community measured hope after years of disappointments. "We've had so many failures," Sung says, "and there's been a lot of heartbreak over many years in this community." Other researchers also praised the development. "This news has really buoyed everyone's expectations of what might be possible," says Rachel Harding, a toxicologist at the University of Toronto. Harding, who wasn't involved in the trials, researches the underlying cause of Huntington's.