12 ms·

What part of this discovery was made thanks to NIH and/or NSF funding from the USA, or the NIHR in the UK? I don't ask to strictly bring up politics, but inste

What part of this discovery was made thanks to NIH and/or NSF funding from the USA, or the NIHR in the UK? 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/ https://pmc.ncbi.nlm.nih.gov/articles/PMC5468112/ 2. https://www.scotusblog.com/2025/08/supreme-court-allows-trump-administration-to-terminate-783-million-in-nih-grants-linked-to-dei-initiatives/ https://www.scotusblog.com/2025/08/supreme-court-allows-trum...

amelius 1y ago
It's standing on the shoulders of giants, and the one on top gets to reap all the benefits.
- mattkrause 1y ago
  Even that quote is a bit of a disservice to modern science: it's a massive pyramid made of thousands and thousands of individual contributions, including many bits of deep background and outright "failures". 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.
jmcgough 1y ago
> Scientists need better messaging or else we're going to stop having breakthroughs like this 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.
- mattkrause 1y ago
  This is where the funders really ought to step up. 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.
- drstewart 1y ago
  [flagged]
  - conception 1y ago
    You missed the second part of that sentence buddy. Your examples would match if every base commander had to write public messaging on their bases’ budgets and projects.
    - derektank 1y ago
      Every base commander regularly meets with their Public Affairs personnel and will often attend public events in the local region their base is in. The funding is not directly tied to their public perception, sure, but there is a causal chain that every O-6 and above is well aware of.
  - xphos 1y ago
    > No, it's not sad that you need to justify the use of public money. You don't ask your plumber which computer network you should build for a fortune 500 company for the same reason I don't ask a computer programmer how to fix leaky pipes. People who study in an area actually have much stronger basis for having opinions rather than keyboard warriors who are upset that there mythological studies have been debunked time and time again. That's not science that is cult behavior. If you want to influence that decision process work in that field and provide justifications for that vaccine research and some new angle that's been missed. The American public is not a intelligent member of the medical community there opinion should not have the same weight in day to day operations as the medical community. They can allocate an amount we want to do research and should ask the NIH to do board research because that is effective and has an overwhelmingly strong record having done the background research for basically ever medical advancement made for a long long time. So, no I think we should allow a panel of experts to evaluate what is worth funding in research. Give the NIH a budget to hire a panel of field experts like they have been for a long time and fund research that panel says is worth it. Autism and vaccine linkage is studied and has been shown several times to have no strong correlation with vaccination. The idea we need to study that more is stupid and experts say it is stupid because it steals funding from actual research into other environmental factors that haven't been studied yet. Maybe its PFAS chemicals maybe its something else.
oulipo2 1y ago
I'm pretty sure some of it is direct funding by governmental agencies, but even if that wasn't the case, all the basis of the theory, and the groundwork was laid by researchers and universities using those grants. You need public money for a healthy society
sleight42 1y ago
There's little money to be made with HD. It's a 1 in 30,000 disease. There's been little reason for anyone other than state sponsors to support its treatment. Add this to the reason's to be disgusted by capitalism. Spoken as a widower of an HD wife.
- s5300 1y ago
  [dead]
- missedthecue 1y ago
  I'm sorry to hear about the passing of your wife. How common a disease is doesn't have much to do with treatment efforts. Cystic Fibrosis has practically been cured by Big Pharma and only ~40,000 people in the entire US have it.
  - pkaye 1y ago
    That is because the Cystic Fibrosis foundation funded some of the research and drug development to make the first treatment possible. They had to essentially operate in a VC model to get the treatments that these CF patients need. https://www.cff.org/about-us/our-venture-philanthropy-model https://www.cff.org/about-us/our-venture-philanthropy-model
    - missedthecue 1y ago
      Most disease categories have advocacy and funding groups, with varying amounts of success. The point is that rare diseases get new treatments all the time from private for-profit firms. In fact, orphan diseases are extremely profitable, and the FDA offers fast tracked approval decisions, making them an enticing niche to focus on.
- kulahan 1y ago
  It really has nothing to do with capitalism. There are special grants in the US for researching rare diseases, specifically to ensure money isn't the barrier. As an aside because I'm pedantic about the language, apostrophes are never used to show pluralism.
melagonster 1y ago
We even do not need to calculate NIH grants; I am pretty sure that all databases that were used here are from NCBI. If there were no NIH, all research would be impossible in modern biology.
stevenwoo 1y ago
The problem is the soundbite of some of these studies on the surface is ridiculous to lay people but even good studies with bad sound bites are used as weapons against science funding in the USA. The shrimp on a treadmill study is still used as argument against science funding today. https://www.npr.org/2011/08/23/139852035/shrimp-on-a-treadmill-the-politics-of-silly-studies https://www.npr.org/2011/08/23/139852035/shrimp-on-a-treadmi...
- privatelypublic 1y ago
  I always thought the shrimp was a random meme. This is even better! My understanding is There's also studies of "duh!" Things, so theres a paper to cite instead of an assumption.
caycep 1y ago
I think it is a UK study. It actually has been going on for a number of years, I've seen one of the PIs giving a number of big conference talks (Tabrizi) for a while

amelius 1y ago

It's standing on the shoulders of giants, and the one on top gets to reap all the benefits.

mattkrause 1y ago
Even that quote is a bit of a disservice to modern science: it's a massive pyramid made of thousands and thousands of individual contributions, including many bits of deep background and outright "failures". 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.

jmcgough 1y ago

> Scientists need better messaging or else we're going to stop having breakthroughs like this 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.

mattkrause 1y ago
This is where the funders really ought to step up. 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.
drstewart 1y ago
[flagged]
- conception 1y ago
  You missed the second part of that sentence buddy. Your examples would match if every base commander had to write public messaging on their bases’ budgets and projects.
  - derektank 1y ago
    Every base commander regularly meets with their Public Affairs personnel and will often attend public events in the local region their base is in. The funding is not directly tied to their public perception, sure, but there is a causal chain that every O-6 and above is well aware of.
- xphos 1y ago
  > No, it's not sad that you need to justify the use of public money. You don't ask your plumber which computer network you should build for a fortune 500 company for the same reason I don't ask a computer programmer how to fix leaky pipes. People who study in an area actually have much stronger basis for having opinions rather than keyboard warriors who are upset that there mythological studies have been debunked time and time again. That's not science that is cult behavior. If you want to influence that decision process work in that field and provide justifications for that vaccine research and some new angle that's been missed. The American public is not a intelligent member of the medical community there opinion should not have the same weight in day to day operations as the medical community. They can allocate an amount we want to do research and should ask the NIH to do board research because that is effective and has an overwhelmingly strong record having done the background research for basically ever medical advancement made for a long long time. So, no I think we should allow a panel of experts to evaluate what is worth funding in research. Give the NIH a budget to hire a panel of field experts like they have been for a long time and fund research that panel says is worth it. Autism and vaccine linkage is studied and has been shown several times to have no strong correlation with vaccination. The idea we need to study that more is stupid and experts say it is stupid because it steals funding from actual research into other environmental factors that haven't been studied yet. Maybe its PFAS chemicals maybe its something else.

oulipo2 1y ago

I'm pretty sure some of it is direct funding by governmental agencies, but even if that wasn't the case, all the basis of the theory, and the groundwork was laid by researchers and universities using those grants. You need public money for a healthy society

sleight42 1y ago

There's little money to be made with HD. It's a 1 in 30,000 disease. There's been little reason for anyone other than state sponsors to support its treatment. Add this to the reason's to be disgusted by capitalism. Spoken as a widower of an HD wife.

s5300 1y ago
[dead]
missedthecue 1y ago
I'm sorry to hear about the passing of your wife. How common a disease is doesn't have much to do with treatment efforts. Cystic Fibrosis has practically been cured by Big Pharma and only ~40,000 people in the entire US have it.
- pkaye 1y ago
  That is because the Cystic Fibrosis foundation funded some of the research and drug development to make the first treatment possible. They had to essentially operate in a VC model to get the treatments that these CF patients need. https://www.cff.org/about-us/our-venture-philanthropy-model https://www.cff.org/about-us/our-venture-philanthropy-model
  - missedthecue 1y ago
    Most disease categories have advocacy and funding groups, with varying amounts of success. The point is that rare diseases get new treatments all the time from private for-profit firms. In fact, orphan diseases are extremely profitable, and the FDA offers fast tracked approval decisions, making them an enticing niche to focus on.
kulahan 1y ago
It really has nothing to do with capitalism. There are special grants in the US for researching rare diseases, specifically to ensure money isn't the barrier. As an aside because I'm pedantic about the language, apostrophes are never used to show pluralism.

melagonster 1y ago

We even do not need to calculate NIH grants; I am pretty sure that all databases that were used here are from NCBI. If there were no NIH, all research would be impossible in modern biology.

stevenwoo 1y ago

The problem is the soundbite of some of these studies on the surface is ridiculous to lay people but even good studies with bad sound bites are used as weapons against science funding in the USA. The shrimp on a treadmill study is still used as argument against science funding today. https://www.npr.org/2011/08/23/139852035/shrimp-on-a-treadmill-the-politics-of-silly-studies https://www.npr.org/2011/08/23/139852035/shrimp-on-a-treadmi...

privatelypublic 1y ago
I always thought the shrimp was a random meme. This is even better! My understanding is There's also studies of "duh!" Things, so theres a paper to cite instead of an assumption.

caycep 1y ago

I think it is a UK study. It actually has been going on for a number of years, I've seen one of the PIs giving a number of big conference talks (Tabrizi) for a while