A glimmer of hope in the treatment of Huntington's disease
When the news broke on 24 September 2025 of a major breakthrough in the treatment for Huntington's disease, there was at last a glimmer of hope for the Huntington’s community.
Understandably, many questions have followed. We aim to answer some of them.
What is the study?
uniQure has been testing a treatment called AMT-130 in clinical trials since 2019. People are recruited onto clinical trials through Enroll-HD. The clinical trial for this drug has now closed, so it is no longer possible to join.
In announcing the results from three years of treatment, researchers reported that AMT-130 was safe, well tolerated, and slowed progression of Huntington’s disease by around 75% as measured using the composite unified Huntington’s disease rating scale (cUHDRS).
What does this mean?
The findings are based on fewer than 30 participants, only some of whom received the high dose that showed the greatest benefit. As said in the HD Buzz article:
“All of the findings were compared to data from Enroll-HD, a natural history control sample, allowing scientists to judge whether treated participants were declining more slowly than expected if they weren’t taking the drug.”
For a clear explanation of what this means, you can read the detailed analysis on HD Buzz:
The HD Buzz article also addresses questions about earlier reported side effects and how these were overcome.
What is AMT-130?
AMT-130 is a gene therapy treatment, delivered as a single injection directly into the brain during a neurosurgical operation lasting more than 12 hours. Because of the way it works and its complexity, the procedure may not be suitable for everyone.
The goal of this treatment is to reduce the levels of the toxic protein permanently in a single dose (BBC News)
This is not a cure for Huntington’s disease, but it represents the most promising clinical trial results to date. Other pharmaceutical companies are also working on additional potential treatments.
As said by Cath Stanley BEM, Chief Executive of the Huntington’s Disease Association
“The news last week was remarkable. We now have to understand what this means. The data we have seen is from 29 participants in the trial. Even if the drug is approved by the necessary regulatory bodies, the final decision on whether it is available on the NHS could take several years. We also need to be realistic: surgery lasting more than 12 hours will not be suitable for everyone. We have already lost too many to this disease, and this treatment may come too late or not be suitable for others. It is, however, a huge step forward in treatment for Huntington’s disease. We remain committed to supporting everyone affected by this disease."
Have there been side effects?
In the press release by uniQure it says:
“As of June 30, 2025, no new drug-related serious adverse events have been observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure, which all resolved.”
Earlier stages of the trials did show some side effects, this is mentioned in the HD Buzz article.
Should I get tested for Huntington’s disease now?
This research should not influence your decision to be tested for Huntington’s disease. It
remains a very personal decision. It should not be assumed that it will improve your access to this treatment, as this has not been suggested by anyone.
If you are considering testing, support is available through our website or by speaking with your specialist adviser.
Can I get access to this treatment?
Not yet. Approval is still some way off. The process will involve regulatory reviews in order: first the FDA (USA), then the EU, then the MHRA (UK). The final decision on NHS availability will be made by NICE for England and possibly the All Wales Medicines Strategy Group (AWMSG).
This process could take several years.
Prof Paresh Malhotra, Professor of Clinical Neurology, Imperial College London, said:
“This announcement represents a potential major step in the treatment of Huntington’s Disease and Neurodegeneration more broadly. The treatment consists of a highly advanced approach involving neurosurgery and gene therapy, and it appears to have significant effects on disease slowing when treated patients were compared to a control group from another cohort of people with Huntington’s. This is a company announcement and the results have not yet been presented at a scientific conference or published in a journal, but they will bring hope to people affected by this devastating disease, and provide further evidence that better understanding of disease mechanisms will bring us closer to effective treatments.”
What happens next?
We will continue to share updates as soon as they are available. As a charity, we are committed to striving to ensure that any treatments developed are accessible to everyone who needs them.
We need your support more than ever to help us continue supporting those affected by Huntington’s disease.
Please note this information is only relevant for England and Wales.