ICYMI: Novel Gene Therapy Offers New Hope for Slowing Huntington’s

Huntington’s is a rare, fatal neurodegenerative disease driven by deteriorating neurons in the brain. Although patients can seek interventions to manage their symptoms, there are no approved treatments to slow or stop them, and most people die within one to three decades after signs of the disease begin. But a small clinical trial involving an experimental gene therapy could pave the way for a first-of-its-kind intervention to slow Huntington’s progression.

The disease is caused by a mutation in the huntingtin gene. Everyone possesses this gene, but the mutation causes people with Huntington’s to produce a harmful version of the huntingtin protein, which causes their neurons to break down and die. The therapy, called AMT-130 and developed by the company uniQure, is delivered via a lengthy, one-time neurosurgery. It involves using a harmless virus to deliver a specific piece of DNA into the brain. This DNA was designed to ultimately instruct neurons to stop producing the faulty huntingtin protein.

Big Picture: An estimated 41,000 people in the United States are living with Huntington’s today, and more than 200,000 people nationally are at risk of developing it, according to the Huntington’s Disease Society of America. The clinical trial involved 29 patients, 17 of which received a high dose of AMT-130 and 12 of which received a low dose. Twelve people in the high dose category were followed over the course of three years, and their symptom progression slowed by 75% compared to people who did not receive any treatment. The results of the trial were reported by uniQure, and they have yet to be peer reviewed. But experts say this breakthrough offers hope that the therapy could one day gain regulatory approval and become available to patients.

Read More: In a first, a gene therapy seems to slow Huntington disease. Science

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About the Author

Bella Isaacs-Thomas

Bella is the Staff Writer and Editor for BrainFacts. She previously worked as a digital science reporter at PBS NewsHour, where she was named a fellow in the 2022-2023 class of the National Science-Health-Environment Reporting Fellowships (SHERF) and earned a 2021 D.C. Science Writers Association Newsbrief Award for writing. She graduated from the University of Michigan with a bachelor's degree in 2018.