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Huntington's disease treated for first time

bbc.com

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Re: Huntington's disease treated for first time

#71

Post by lawlessone »

ericmcer wrote: post #66
ksajadi wrote: post #24

This is off topic, slightly but I think a good place to say this: 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.

This is disingenuous. There has been a strong push back against illegal-immigration in the west. The media has completely reframed the discussion to "How can they be opposed to immigration" because if they said "how can they be opposed to illegal-immigration" their argument would fall apart pretty quickly. No one with a brain is arguing that immigration doesn't provide tremendous value.

>How can they be opposed to immigration" because if they said "how can they be opposed to illegal-immigration" their argument would fall apart pretty quickly.

Isn't this also disingenuous? A significant proportion of the groups against immigration are against any immigration and have been floating trial balloons for "remigration" for non-white citizens.

Re: Huntington's disease treated for first time

#72

Post by lostlogin »

> In the UK, the NHS does pay for a £2.6m-per-patient gene therapy for haemophilia B.

A misleading data point. This group of people were treated so poorly by the state that something had to be done. I don’t think this is setting a benchmark.

https://haemophilia.org.uk/public-inquiry/the-infected-blood...

Re: Huntington's disease treated for first time

#73

Post by jjtheblunt »

kulahan wrote: post #64
sleight42 wrote: post #37

Earlier quoted context omitted.

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.

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.

> apostrophes are never used to show pluralism.

in Dutch they are for some words, e.g., 1 ski, 2 ski's. i have no idea how that arose historically.

Re: Huntington's disease treated for first time

#74

Post by karmelapple »

kulahan wrote: post #64
sleight42 wrote: post #37

Earlier quoted context omitted.

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.

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.

I am scared that special grants to research rare diseases will go away, too.

If we're trying to figure out what the most benefit for each taxpayer dollar is, then a rare disease won't win out over, say, cancer research.

Someone may consider researching a rare disease as "waste," even though to everyone including the previous poster who is a widow because of HD, it is far from a waste.

When there is not much of a profit motive to do something - whether going to the moon or fighting a rare disease - public money is the best way to do it. And even throwing a fairly small percentage at it can create big achievements.

And that's one reason I'd like to see how much money and time went into this. We might be surprised that it's fairly small in the grand scheme of biomedical research costs!

Re: Huntington's disease treated for first time

#75

Post by Shortness8 »

lostlogin wrote: post #72

> In the UK, the NHS does pay for a £2.6m-per-patient gene therapy for haemophilia B. A misleading data point. This group of people were treated so poorly by the state that something had to be done. I don’t think this is setting a benchmark. https://haemophilia.org.uk/public-inquiry/the-infected-blood...

Don't often comment on HN but have to point this out as a med student in the UK: the cost-benefit roughly works out for those in favour of giving the therapy when the alternative is a lifetime of coming to hospital 3 times a week for Factor IX infusions, and the additional cost of stays in hospital for bleeds/haemarthroses and the complications thereof. Of course, this also ignores the human cost, particularly the extra care/stress around avoiding cuts/bruises in every aspect of life. In this respect these gene therapies appear lifechanging for those who suffer from the disease. [1]

I will also say I know the team who wrote the guidelines for use of these therapies. I believe they were mostly finished before the infected blood scandal became a big story. Politics didn't come into it.

[1]: https://www.bbc.co.uk/news/articles/c4nnn51rdrzo

Re: Huntington's disease treated for first time

#76

Post by kulahan »

jjtheblunt wrote: post #73
kulahan wrote: post #64

Earlier quoted context omitted.

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.

> apostrophes are never used to show pluralism. in Dutch they are for some words, e.g., 1 ski, 2 ski's. i have no idea how that arose historically.

The Nords are a pox upon us all. TIL though, thanks - I probably should've been clear I only meant in English :)

Re: Huntington's disease treated for first time

#78

Post by AudiomaticApp »

sleight42 wrote: post #40
petesergeant wrote: post #2

More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > 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-…

It always seemed that an mRNA treatment was going to be the way forward for treating HD, speaking as an HD widower. And here my government is actively working to suppress mRNA therapies because of fucking politics. Fuck them.

This isn't an mRNA treatment. The m in mRNA stands for "messenger", not micro. mRNA covid vaccines don't mess with the genome anything like this treatment does, and indeed, this treatment is much more dangerous than any vaccine would be.

Re: Huntington's disease treated for first time

#79

Post by cogman10 »

w10-1 wrote: post #69

(non-expert) This is promising but needs publication and expert review. Here's the actual company statement from today: https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-positive-topline-results-pivotal-phase-iii There's also a June 2024 article: https://www.cgtlive.com/view/huntington-disease-gene-therapy-nets-rmat-designation That explains a bit more: (1) neuro-surgery introduces gene…

If the approach works, wouldn't this also be really good news for other progressive generic disorders?
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