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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

#61

Post by mjburgess »

lkey wrote: post #57
mjburgess wrote: post #32

Earlier quoted context omitted.

This seems helpful, but I think the misattribution of a general "anti-immigrant" sentiment to immigration detractors is part of the problem. 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 deniali…

"Very few detractors in the west have any issues with highly qualified immigrants occupying scientific or research roles." Laughable, these 'few' reside in the highest seats of power and exist among the 'advisors' to the cabinet. "It's a radical kind of dismissiveness" People keep telling me I'm wrong in the wrong way, if they were nicer I'd consider their opinions. This is an irrational position to stake out, it sho…

I personally don't care about immigration. I have yet ever to be negatively affected by it, and have tended to find immigrants more pleasant than my own citizens. I'm more worried about our aging, obsese, immobile populations who arent breeding, and need 3-to-1 worker ratios to support, that we cannot provide. I am, personally, relatively open borders -- for at least that reason. I am more motivated by welfare demands and geopolitics, both of which benefit from larger active populations in our borders.

However, what I want doesn't really matter. It's a democracy, and half of a country will not be ignored. You either listen and find some way to give them what they want in the least objectionable way; or they will take what they want, and take revenge on you for your obstinacy. They will do to you what you insist on doing to them: not listening to you at all as they enact the most extreme form of policies which they agree with.

You speak as if your views express those of a powerful majority who will impose them on your detractors. This a dangerous power fantasy. The castle has been ransacked, the draw bridge is down, and you've no army left.

You either draw up terms of surrender, or they will be drawn up for you.

Re: Huntington's disease treated for first time

#62

Post by missedthecue »

sleight42 wrote: post #37
karmelapple wrote: post #16

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 laun…

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.

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.

Re: Huntington's disease treated for first time

#63

Post by AnimalMuppet »

mjburgess wrote: post #61
lkey wrote: post #57

Earlier quoted context omitted.

"Very few detractors in the west have any issues with highly qualified immigrants occupying scientific or research roles." Laughable, these 'few' reside in the highest seats of power and exist among the 'advisors' to the cabinet. "It's a radical kind of dismissiveness" People keep telling me I'm wrong in the wrong way, if they were nicer I'd consider their opinions. This is an irrational position to stake out, it sho…

I personally don't care about immigration. I have yet ever to be negatively affected by it, and have tended to find immigrants more pleasant than my own citizens. I'm more worried about our aging, obsese, immobile populations who arent breeding, and need 3-to-1 worker ratios to support, that we cannot provide. I am, personally, relatively open borders -- for at least that reason. I am more motivated by welfare demand…

> The castle has been ransacked, the draw bridge is down, and you've no army left.

Well, no, not that either. You're making the same mistake from the other side. The side you say has "no army left" also is half the country. And that half of a country will not be ignored either.

Both sides demand to be listened to, but neither side is willing to compromise or to admit defeat, even temporarily.

So I expect that power will continue to swap back and forth, that when in power both sides will do their best to implement their plans, and when they are out of power they will suffer the other side's vengeance for their obstinacy. I don't see it improving soon. I suspect we'll see at least another two election cycles of this viciousness.

Re: Huntington's disease treated for first time

#64

Post by kulahan »

sleight42 wrote: post #37
karmelapple wrote: post #16

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 laun…

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.

Re: Huntington's disease treated for first time

#65

Post by mjburgess »

AnimalMuppet wrote: post #63
mjburgess wrote: post #61

Earlier quoted context omitted.

I personally don't care about immigration. I have yet ever to be negatively affected by it, and have tended to find immigrants more pleasant than my own citizens. I'm more worried about our aging, obsese, immobile populations who arent breeding, and need 3-to-1 worker ratios to support, that we cannot provide. I am, personally, relatively open borders -- for at least that reason. I am more motivated by welfare demand…

> The castle has been ransacked, the draw bridge is down, and you've no army left. Well, no, not that either. You're making the same mistake from the other side. The side you say has "no army left" also is half the country. And that half of a country will not be ignored either. Both sides demand to be listened to, but neither side is willing to compromise or to admit defeat, even temporarily. So I expect that power w…

Maybe. Certainly if detractors go to excess then they'll breed resentment from the supporters, and round and round.

At the moment though, i think there's lots of people speaking as if it were 2022 and all of that ammunition has now been expended and the stores are bare

Re: Huntington's disease treated for first time

#66

Post by ericmcer »

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.

Re: Huntington's disease treated for first time

#67

Post by pkaye »

missedthecue wrote: post #62
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.

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.

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

Re: Huntington's disease treated for first time

#68

Post by missedthecue »

pkaye wrote: post #67
missedthecue wrote: post #62

Earlier quoted context omitted.

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.

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

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.

Re: Huntington's disease treated for first time

#69

Post by w10-1 »

(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-virus to putamen and caudate nucleus; (2) virus delivers gene that produces micro-RNA; (3) the micro-RNA blocks the messenger RNA of the bad gene, reducing bad protein production.

The 2023 study is said to have 39 patients (BBC and their recent statement reports 29). The reported findings may be significant but seem small (e.g., low dose: 0.39 of 14.1 points). Earlier they reported composites from the Unified HD Rating Scale, which has the usual caveats for the behavioral and functional sub-measures (vs. the more reliable motor and cognitive). Today's statement instead focuses on the more objective measures instead of the composite.

Earlier, high-dose responders reportedly didn't just stabilize but got better -- unclear how. The more recent findings report that the disability still progressed, but slowed relative to "propensity-matched" controls. (Note 4 of 10 controls opted to join the trial after 12 months.)

Both mention improvement in NfL (neurofilament light chains), which is an objective but nonspecific (and highly variable) measure of the degree of neuronal decomposition. The statement quantifies this at ~8% -- unclear if this level is convincing.

For such an invasive treatment for a slowly progressing, relatively rare disease, they're probably gathering and publishing data as fast as possible. The short-term results seem good, and it will be good to see long-term results over time.

It's possible some effect seen is due e.g., to immune adjuvants or something else during the therapy, and I would want experts to review the propensity matching.

I would be concerned that the micro-RNA produced either also binds with epitopes from other messenger RNA or induces some immune response. Remember, there's no reversal agent or half-life elimination for such genetic treatments.

So: room for hope but also for caution.

Re: Huntington's disease treated for first time

#70

Post by lawlessone »

siva7 wrote: post #21

Medical progress has been insane in the last few years through technological breakthroughs. It's not out of reach to think that most types of cancers will be curable 20 years from now on.

it's great, it feels like the first mRNA vaccine opened a dam.
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