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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

#81

Post by senkora »

kennywinker wrote: post #56
trallnag wrote: post #52

"If one of your parents has Huntington's disease, there's a 50% chance that you will inherit the altered gene and will eventually develop Huntington's too." Have they never heard of genetic diagnostics? For example with a combination of preimplantation generic testing and in-vitro fertilization you can prevent passing on known genetic mutations to the next generation.

While technically possible, that option isn’t really available to most people. Tho I suppose 17 hour brain surgery with gene therapy is also not available to most people, so fair enough. Anyway, they’re just describing the heritability of the disease there.

An optimal society would provide no-cost embryo screening and IVF to couples with the gene for HD, because that cost is certainly far I hope that we can work towards such a society.

(And of course this research is still worth doing to help the population who already have the illness)

Re: Huntington's disease treated for first time

#82

Post by presidentender »

One of my mom's best friends when I was a kid had Huntington's. She was a few years older than mom, and her sons were a few years older than my brother and I. One of them chose to get tested. The other chose not to. I remember thinking that was foolish, but I was seven years old. In retrospect, it's strange that a seven-year-old was privy to such things.

Re: Huntington's disease treated for first time

#83

Post by JetSetWilly »

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.

I have a feeling this migrant didn’t get off a dinghy with all the other engineers and scientists so probably isn’t raising a lot of concern for most. Conflating “immigration is too high” with “anyone who thinks immigration is too high is a racist who thinks they are all freeloaders” doesn’t work anymore, no amount of media propaganda will change that.

Re: Huntington's disease treated for first time

#84

Post by tsoukase »

Huntington's is among the best candidates for a genetic cure: well known gene and mechanism, definitive pre symptomatic diagnosis, slow progression.

But I am still reluctant. It's phase 1/2 (ie exploratory) and the phase 3 is the hard part that takes many years. Also it's disease slowing not stopping.

Re: Huntington's disease treated for first time

#85

Post by tomrod »

mbreese wrote: post #19
tomrod wrote: post #4

Quick skimmed, is there a peer reviewed paper?

I don't see one yet -- but the main people mentioned in the article have a long publication record on Huntington's. This trial has been going on for a while and this is an interim media report. I don't think they've reached an endpoint yet. I believe this is the clinical trial they are reporting on: https://clinicaltrials.gov/study/NCT04120493 This trial also appears be open at UCSF...

Awesome

Re: Huntington's disease treated for first time

#86

Post by ourmandave »

edm0nd wrote: post #80
ourmandave wrote: post #77

Has anybody thought about changing the name from Huntington's disease to after the lead person or team that found the cure?

Is that a thing that happens in the medical field? Are we going to rename Polio to The Bill & Melinda Gates Foundation?

Polio is short for Poliomyelitis, but I'd call it Jonas Salk disease.

Re: Huntington's disease treated for first time

#87

Post by missedthecue »

tsoukase wrote: post #84

Huntington's is among the best candidates for a genetic cure: well known gene and mechanism, definitive pre symptomatic diagnosis, slow progression. But I am still reluctant. It's phase 1/2 (ie exploratory) and the phase 3 is the hard part that takes many years. Also it's disease slowing not stopping.

The FDA has already agreed that data collected from this trial could be used to support a future BLA, and UniQure said this morning they're moving ahead with the BLA. In other words, uniQure likely will not need to run another trial before obtaining FDA approval for AMT-130.

Re: Huntington's disease treated for first time

#88

Post by zdragnar »

xphos wrote: post #58
drstewart wrote: post #44

Earlier quoted context omitted.

[flagged]

> 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 tim…

That's a fantastic way to fall victim to grift. Your "panel of experts" can easily be as biased as anyone else.

When you give people vast authority on the basis of their expertise (even assuming the expertise is genuine), anything that challenges it becomes not a novel idea worth exploring, but a direct challenge to their authority.

Planck's principle- that science advances one funeral at a time- is rather apropos here.

Re: Huntington's disease treated for first time

#89

Post by onionisafruit »

presidentender wrote: post #82

One of my mom's best friends when I was a kid had Huntington's. She was a few years older than mom, and her sons were a few years older than my brother and I. One of them chose to get tested. The other chose not to. I remember thinking that was foolish, but I was seven years old. In retrospect, it's strange that a seven-year-old was privy to such things.

I chose not to get tested when the test was new. I still haven’t, but I feel confident I dodged the bullet based on my age vs my relatives’ age of onset. I used to wonder if I could take the news of having HD. Now I wonder how life would have been different knowing for sure I don’t have it.

Re: Huntington's disease treated for first time

#90

Post by privatelypublic »

stevenwoo wrote: post #49
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...

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.

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