Positive news coming from pharmaceutical drug company uniQure today.
In a recent trial, the treatment AMT 130 was safe and well tolerated, and slowed progression of Huntington’s disease by around 75% (Footnote as to how this was measured 1).
As said by Professor Ed Wild,
This result is the good news we've been working and waiting for, not just a treatment that slows progression of this terrible disease, but one that does so with truly stunning effectiveness. It is nothing less than the dawn of a new age for families impacted by Huntington's disease. We must now work diligently to turn this breakthrough into something that benefits everyone who needs it.
AMT-130 is an experimental gene therapy developed by uniQure to slow the progression of Huntington’s disease. Gene therapy aims to fix or replace genes that don't work properly.
AMT130 is administered as a single dose via injection directly into the brain, in a neurosurgical operation lasting over 12 hours. Patients who have been treated also had biomarker changes (a measurable indicator that evaluates the effectiveness of treatments) suggesting that the effect of the drug comes from its ability to rescue neurons (nerve cells). This means the results show that the drug is helping to protect and recue brain cells, which is why it works.
Cath Stanley BEM, Chief Executive of the Huntington’s Disease Association, said,
This is a significant breakthrough, and I am sure it will bring hope to anyone affected by Huntington’s disease. This trial shows an astonishing 75% slowing of progression in the disease. That is remarkable and will bring much needed optimism to the Huntington's disease community.
The trial is now finished.
UniQure’s treatment is going for approval in the USA next year, after that it will go for approval in the EU and MHRA ( the UK drug approval body). The final stage will be with NICE who will decide on its suitability to be delivered through the NHS. Unfortunately, this process will take some time.
However, today we can be reassured that hope is on the horizon. We will share any updates with you as and when we receive it.
We would like to thank everyone from the Huntington's disease community who have taken part in not only this clinical trial but all clinical trials including Enroll-HD. Without you, our researchers and pharmaceutical companies would not be able to share this news with us today. We also want to thank the incredible and passionate teams of researchers who work tirelessly on a wide range of clinical trials, all with the same goal, of finding a treatment to slow down or stop the progression of Huntington's disease.
Footnote 1
The composite Unified Huntington’s Disease Rating Scale (cUHDRS) is a scoring algorithm that combines Total Functional Capacity (TFC), Total Motor Score, Symbol Digit Modalities Test and Stroop Word Reading measures.
FAQs
Can I take part in this trial?
No, the trial is now closed.
Does it help people who have advanced Huntington’s disease?
At this time, the trial has only shown improvements in those with early stages of the disease
Should this affect my decision to get a genetic test?
The drug is several years away from approval. Taking a test is a decision you should make independently and not because you think this may enable you to get the drug.
Can I get access to the treatment?
The treatment has to be approved by a number of regulatory bodies; this may take some time, we will keep you updated as and when this happens.
Can I ask my consultant or GP about access to this drug?
As the trial has now closed, your consultant or GP will not be able to obtain access to this drug for you until it has been approved.
What is the treatment?
The drug is delivered via surgery into the brain. The surgery takes more than 12 hours.
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) you may be offered recruitment for suitable trials.
If you would like to find out more about research or sign up to Enroll-HD, you can do so below.