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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

#41

Post by sleight42 »

didgeoridoo wrote: post #14
CookiesOnMyDesk wrote: post #7

Earlier quoted context omitted.

It’s covered in the article >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.

I don’t think this quite answers the curiosity of whether starting treatment e.g. at birth would virtually eliminate morbidity, or whether it only slows the decline once it has started. 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 lif…

To me, as an HD widower, it would have meant that my dead wife would had lived until 2043 and had a decade more of a mostly normal life.

Re: Huntington's disease treated for first time

#42

Post by sleight42 »

jmclnx wrote: post #26

This needs to be said, with Trump's cutting of College Basic Research Funds, many of these great breakthroughs will occur in other Countries. The US is/was the lead in biotech, it is now giving up its lead. That means one of the US largest industries, employing many with high pay, will shrink, due to US policies. In a few years it may not exist unless funding is restored soon. I personally know people who's grants ha…

Even more: mRNA. Trump is trying to kill mRNA research because he sees it as tied to the politics of COVID.

Good science is not political. Politicians making it so are idiots at best and evil at worst. See also "Hanlon's Razor"

Re: Huntington's disease treated for first time

#43

Post by mattkrause »

bjornsing wrote: post #25
Sebalf wrote: post #12

Earlier quoted context omitted.

The major hurdle of current gene therapies is delivery to the tissue where the defective gene product is causing damage. For instance lipid nanoparticles are only being used to deliver gene therapies to the liver, because if you inject them they just end up there and not much anywhere else. In this case they are using an virus called "adeno asociated virus 5" (AAV5), which does not naturally infect the brain AFAIK. T…

I guess it needs to get across the blood-brain barrier. But that shouldn’t take 10+ hours of surgery, I don’t think.

Surgery can be slow, and brain surgery doubly so.

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.

Re: Huntington's disease treated for first time

#44

Post by drstewart »

jmcgough wrote: post #20
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…

> 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 frien…

[flagged]

Re: Huntington's disease treated for first time

#45

Post by singulasar »

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.

Let's hope the defunding of medical research can stop so this can become true

Re: Huntington's disease treated for first time

#46

Post by Supermancho »

whatsupdog wrote: post #34
Hasz wrote: post #33

Earlier quoted context omitted.

I strongly disagree with this. While I am generally pro-immigration, injecting a political view into an article ostensibly about a new scientific discovery is how science loses credibility and objectivity. See the "trust the science" phrase weaponized during COVID in the US. Let people draw all the inferences they want about the origins of the scientists involved, but a hamfisted paragraph about a.b scientist being a…

Also it sends a message that only scientists are welcome as immigrants. There's millions of immigrants who contribute positively to the society, who aren't scientists.

> Also it sends a message that only scientists are welcome as immigrants.

This is not a conclusion I would make without trying to make an anti-immigrant argument.

Re: Huntington's disease treated for first time

#47

Post by [deleted] »

mjburgess wrote: post #32
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 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…

[deleted]

Re: Huntington's disease treated for first time

#48

Post by stinkbeetle »

mjburgess wrote: post #32
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 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…

This isn't addressing the main point of your comment, but a side issue:

> 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 [...].

I'm not a detractor of any individual immigrant, certainly not a very skilled one. But I am dumbfounded when I hear people say how wonderful immigration is "for the economy", "for the health system", etc., because we can lure all these bright people over from poor countries with offers they can't get at home.

Like... Mozambique needs good doctors and nurses too. Sudan needs good engineers. Syria needs entrepreneurs.

I don't begrudge the immigrant one bit for moving to get more money or a better life or whatever it is that motivates them, and they sure do contribute to the place they move to. But harvesting the best and brightest minds from poor countries on an industrial scale isn't something that sits too well for me at all. The merits and effects could be debated and disagreed, but it certainly requires much deeper thought than just the greed driven "good for my economy, good for my healthcare, good for me" type quips.

I actually think of it as neocolonialism. The most valuable resource in this day and age is people, and wealthy countries are plundering the human capital from the poor ones like they did with resources in previous centuries. Throwing a bit of charity at them whenever the next outbreak or famine or civil war rolls around doesn't make up for it.

Re: Huntington's disease treated for first time

#49

Post by stevenwoo »

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…

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-treadmi...

Re: Huntington's disease treated for first time

#50

Post by s5300 »

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.

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