I believe this is the clinical trial they are reporting on: https://clinicaltrials.gov/study/NCT04120493
This trial also appears be open at UCSF...
I don't ask to strictly bring up politics, but instead to try and address the broad lack of understanding of how medical breakthroughs like this are made.
It's not done just by drug companies. The article says:
> UniQure says it will apply for a licence in the US in the first quarter of 2026 with the aim of launching the drug later that year.
That's true, but that doesn't talk about the tens to hundreds of research papers that have been published over likely decades to make this discovery a reality. And it doesn't talk about how much public money went into this discovery.
Many people reading this article probably have a vague idea that more than just this company was involved, but I feel it is not at all clear to the vast majority of people, since the vast majority of people are not involved in biomedical research.
I wish there was an easy way to figure out how many dollars, how many grants, how many researchers, went into achieving this breakthrough. And that the media would put that into news articles like this. Trace all the citations back a few orders, and I bet you'll find a massive number of NIH and NIHR grants.
There is unfortunately not more massive, bipartisan public outcry in the US over defunding the essential basic research the NIH does... and it's not new to the current administration, since it was attempted to be done back in 2017, too [1].
Scientists need better messaging or else we're going to stop having breakthroughs like this... and the breakthroughs are already going to slow down thanks to things like the $783 million in cuts to NIH grants that the US SCOTUS authorized in August [2].
1. https://pmc.ncbi.nlm.nih.gov/articles/PMC5468112/
2. https://www.scotusblog.com/2025/08/supreme-court-allows-trum...
Biology is tough in that you can't just "reason" your way to success; it often really does require trying something to see if an approach works.
Sure, but it's really sad that scientists need to justify their funding to the public - they already spend so much time justifying it to the NIH and others for funding.
So many people have had their careers jeopardized by finding pulled mid-project. I am really concerned about our research pipeline, because my post-doc friends are all applying to jobs outside the US now.
A spokesperson from (say) NINDS really ought to be shouting to anyone who will listen about how excited they are to see their <many year>, <many dollar> investment in Huntington's pay off.
I'd love it if they highlighted some of the especially "weird" studies that went into this to demonstrate how important fundamental research is and how it goes in unexpected directions.
No, it's not sad that you need to justify the use of public money.
Unless you think it's sad that the military needs to justify their funding to the public to get more. The military could have a $2 trillion dollar budget but people would ask "why!" when it clearly doesn't need to be justified. Agree?
Here's another thing you'll no doubt agree on: we should fund science with no justifications - I say that we need a $100b invested into more research into the link between vaccines and autism. No justification needed, of course.
Yes, the autism+Tylenol thing is infuriating, but there are nobler ways to express that opinion.
I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen)
In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders.
Just a thought.
Very few detractors in the west have any issues with highly qualified immigrants occupying scientific or research roles. Being opportunistic with which kind of immigrants one offers as Good is partly what's aggravating the issue. It's a radical kind of dismissiveness and denialism which is provoking people and ignoring their issues.
The broad western detraction against immigration at the moment is targeted at specific waves of mass immigration with specific compositions that have specific effects on the places those immigrants have landed.
People are primarily concerned about the ability of state, social and corporate institutions to absorb immigrants at this pace and scale without significant zero-sum effects. And, in addition, the significant amount of state support segments of those populations (eg., esp. asylum seekers) have to receive at a time when gov. are under inflationary pressures, debt pressures, etc. and cannot service their own welfare obligations.
Going, "oh but we get good cancer research from immigration!" is so dismissive to these concerns, that the backfire against this messaging is one of the major contributors to people's disaffection.
The idea that people need to be told that there are people who want to immigrate that are in our national interest to absorb, is just plainly absurd. This is uncontroversial and obvious.
Let people draw all the inferences they want about the origins of the scientists involved, but a hamfisted paragraph about a.b scientist being an immigrant from y country does not have a place here.
But, glad to see other Countries funding their research. I wonder in face of one of the largest blunders made by the US, are they increasing funds ?
Good science is not political. Politicians making it so are idiots at best and evil at worst. See also "Hanlon's Razor"
I lost my first wife to HD in 2013. She was one of the lucky few whose optimism and love of humanity stayed with her through to the end.
If there is a God, I'll never accept it as "loving" as HD is as cruel a slow torture as it comes.
petesergeant•2h ago
> AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT)
and the actual announcement: https://uniqure.gcs-web.com/news-releases/news-release-detai...
> 75% slowing of disease progression as measured by Unified Huntington’s Disease Rating Scale (p=0.003)
> 60% slowing of disease progression as measured by Total Functional Capacity (p=0.033)
> 88% slowing of disease progression as measured by Symbol Digit Modalities Test (p=0.057)
> 113% slowing of disease progression as measured by Stroop Word Reading Test (p=0.0021)
> 59% slowing of disease progression as measured by Total Motor Score (p=0.1741)
basisword•2h ago
CookiesOnMyDesk•2h ago
>It means the decline you would normally expect in one year would take four years after treatment, giving patients decades of "good quality life", Prof Sarah Tabrizi told BBC News.
>The first symptoms of Huntington's disease tend to appear in your 30s or 40s and is normally fatal within two decades – opening the possibility that earlier treatment could prevent symptoms from ever emerging.
didgeoridoo•1h ago
Consider that the disease typically manifests in your 30s — does this mean it would begin 4x later (and thus basically never manifest), or that your 15 year progressive decline from ~35-50 would take 4x longer (giving you a normal lifespan, albeit perhaps with some limitations in your later years)?
sleight42•5m ago
bjornsing•2h ago
JoshTriplett•2h ago
adcoleman6•2h ago
bjornsing•1h ago
devilbunny•44m ago
Sebalf•1h ago
I don't know the details of why AAV5 in particular is their vector of choice in this case, but for whatever reason thats what they've gone with. AFAIK there are no viral or other vectors that consistently infect all brain tissue when injected/ingested, so maybe that's just the best option available. Anyways, it seems that in order to get it to the actual brain tissue that is damaged by the huntington protein (all of it? One particular area?), the best way is to inject it where it needs to go. If you could just pump it into the CSF that would perhaps make things a little bit more tolerable, seeing as you could then just do a spinal tap and inject it that way, but apparently that doesn't work. Or maybe a generalized AAV5 infection has more side effect then targeted injections. Just speculating here.
bjornsing•56m ago
mattkrause•3m ago
The brain is slightly elastic, so you'd want to advance a needle glacially slowly (microns/second) into it so it ends up at the right position. The injection itself is also done slowly (microliters/minute) so you don't cause pressure damage.
They might also do some intraoperative imaging (some ORs have MRI or CT machines), which slows things down, and of course there's tons of cleaning and repair work afterward.
jmcgough•1h ago
saretup•1h ago
nicoburns•1h ago
tkfoss•1h ago
sleight42•7m ago
And here my government is actively working to suppress mRNA therapies because of fucking politics. Fuck them.