CEO Statement on recent Huntington’s disease research updates
Encouraging progress in global Huntington’s research
Last week, we received encouraging news from uniQure about their experimental gene therapy, AMT-130, which has shown potential to slow the progression of Huntington’s disease. This trial is still in its early phase and currently underway in the UK, so it is not yet available in Australia. However, the results offer long-awaited hope for families impacted by HD and pave the way for further research and other companies pursuing clinical trials with huntingtin-lowering drugs.
This marks an important breakthrough. For the first time, a treatment for HD has demonstrated statistically significant slowing of disease progression on clinical measures. It strengthens the case that lowering the huntingtin protein is a valid strategy for treating HD and offers hope that other approaches and trials may also succeed.
This is the news our community has been waiting to hear and it brings much-needed optimism for the future. Huntington’s Australia is following the research closely, sharing updates with the community, and working with researchers and companies to bring trials and new treatments to Australia.
Understanding the Impact: Q&A with Huntington’s Australia
Following the announcement, our CEO answered some common questions from the community.
Can I take part in this trial?
No. Unfortunately this trial is not available in Australia.
What does this gene therapy trial (AMT-130) mean for people with Huntington’s?
This is the first time a treatment for HD has shown statistically significant slowing of disease progression. It provides real optimism and gives weight to other huntingtin-lowering therapies currently in development. If AMT-130 can slow progression by reducing huntingtin protein levels, it validates this approach as a potential treatment and suggests that other similar therapies may also succeed.
Is there a huntingtin lowering drug trial in Australia?
Yes. Skyhawk Therapeutics’ Phase 1 study taking place here in Australia. Their recent update suggested that SKY-0515 can lower the huntingtin protein, as well as PMS1 — another protein that may play a role in driving Huntington’s disease. If you are interested in participating in this trial or would like to find a site near you please visit the Clinical Trials website or email admin@huntingtonsaustralia.au.
How do I get on future trials like this?
The best place to start is by registering with Enroll-HD. Once you are on the Enroll-HD trial (this consists of yearly cognitive and motor tests), researchers will be able to identify who is study-ready and eligible in meeting criteria for future trials.
How have Australians reacted to the news?
Every research update brings hope to families impacted by HD. At the same time, reactions are mixed. For some, this news feels like it has come too late, while others remain eager to see how the trial progresses. What is clear is that each advance brings us closer to treatments becoming available in Australia.
Looking Ahead
While these trial results are encouraging, there are still many steps before treatments like AMT-130 could become widely available. Huntington’s Australia will continue to monitor global research developments, share updates with the community, and advocate for access to trials and new treatments in Australia.
We remain committed to supporting families living with HD, providing timely information, and ensuring hope for the future is grounded in real scientific progress.
Additional Resources
