Huntington's Disease - hope at last?

[SpidersAreShitheads]

The BBC is currently leading with this story:

https://www.bbc.co.uk/news/articles/cevz13xkxpro

For anyone not familiar with it, Huntington's is a bastard of a disease. My dad died from it, and I have a 50% chance of developing it. I've not been tested for the gene as until now, there wasn't really any point as there were no effective treatments.

So this is huge, wonderful news for anyone who's affected by this disease or has the shadow of it hanging over them. The focus is on making this treatment available in the US in the first quarter of 2026, with discussions with the UK to come after.

But - and you probably know what I'm going to say - cost.

It's pioneering neurosurgery that takes around 12-18 hours. The BBC don't give an idea of the cost but they say it's expensive and won't be available for everyone. The BBC does go on to give an example of a treatment the NHS funds that costs £2.6 million per patient.

I'm nearly 50 and fingers crossed, I haven't developed any symptoms yet so hopefully I might not ever need it. I'm not out of the woods yet though. But to know there's a treatment with what looks like an amazing success rate that's available but not affordable, it would be absolutely soul-destroying to anyone who needs it.

I'm probably too close to the subject to think objectively but I would say the same for any other condition where there's treatment that has such a strong outcome. Especially when you then need to balance up the initial outlay against the cost of ongoing care if treatment wasn't given.

I haven't added a poll as I think it's too nuanced but I'd like to think that treatments like this weren't restricted to only those who could afford it privately. I have no idea how the NHS would be able to afford it though.

Such good news. Some hope at last. Best wishes to all the families affected.

The researchers found that patients receiving the treatment experienced 75% less progression of the disease overall, compared to a matched cohort of people with Huntington’s who were not receiving the treatment.
This is the first time a drug trial has reported continuing, statistically significant slowing of Huntington’s progression.
uniQure plans to submit an application to the US Food and Drug Administration early next year requesting accelerated approval to market the drug, with applications in the UK and Europe to follow.

Amazing if successful.

I would also expect/hope that the treatment would not be limited to those who could pay. Although for most of the world for so many things that is the state of things

costs often fall quickly though as the processes get better

This gives hope for so many.
science is amazing

It’s not a cure but a slowing down of the disease. Which is still brilliant.
Will be interesting to see how they get on with the research of preventing it for those that have the gene.

It's a good step but it's only going to buy 3 years. I hope someone can take this reduce the cost and the duration of treatment

It’s wonderful news, it may be too late for a close family friend, however it is a small bit of hope for those affected by it. It is a horrible disease and it’s awful for every person that comes close to it. This is what NHS money should be spent on IMO. So so pleased there is some light at the end of the tunnel.

Its wonderful news, all those people who have been living in the shadow of developing the disease, probably having watched a parent succumb, it must be such a happy day.

If you can listen to it there was an interview on Radio 4 yesterday on the World at one programme.

@Ghhbiuji think you have misunderstood. It will buy people a hell of a lot more than 3 years!

It's wonderful news. I don't know anyone with the disease and I'm a UK taxpayer. I would 100% support this being available on the NHS and would sign petitions and write to my MP to help campaign for it. Just sharing that because OP wasn't sure how people would feel about the cost. I'm delighted for you.

This was such brilliant news today. The team behind this breakthrough must be absolutely on cloud nine. Imagine finding a treatment for something as bleak as this disease, and as cruel.
I have some family experience of the sceptre of this disease hanging over people. They always said getting the test was like Russian roulette, but 3 times more risky.

I genuinely can’t imagine any humane society that lets people die from this terrible disease whilst there is a treatment however expensive.

People diss the NHS but they have funded very expensive orphan treatments in the recent past so I would hope that would continue.

I actually teared up when reading the news today.

To think that in my lifetime we might see the end of this awful disease is such a ray of light.

This is such good news, there's really no words to describe the hope we feel here today.
My dh was lucky to test negative but unfortunately has many family members affected or at risk. I can't describe how devastating this illness is but finally the tireless work of dedicated scientists will help halt it in its tracks.

Because there is a budget, if they pay for one very expenses thing then thousands die of something else. Public health decisions are brutal.

This is only going to get worse with an aging population

SpidersAreShitheads, you and I are almost in exactly the same boat. I lost my dear dad in 2013 to this horrific disease. I am now 50, not tested and unsymptomatic as yet (although every dropped item, brain-fog moment etc makes me think the worst...this is the curse of it). I am SO HAPPY to see this news and I am so grateful to the professors who worked towards this. Prof Tabrizi actually met my dad in the early 2000s and we could see that there was great work going on even then. I really hope that this will be able to help current and future sufferers of HD and also that this research might help get treatment for other terrible diseases like Parkinson's and MND. I couldn't believe it when I saw the story last night. Hope at last and a piece of amazing news. Sending you hugs xx

It’s amazing news. My cousin died from juvenile Huntington’s at just 21. He was the last of that line, the oldest living to 46. Absolutely cruel disease.

Bumping this to say a member came over today to share their positive test results. Obviously its still such a blow but today compared to previous occasions we all had a little more hope that this result won't be the same for this next generation as previous ones before were.

Yes, of course. Health economics is a brutal business. But before, this was an automatic abd often brutally quick death sentence. Now people affected have some hope. And the scientists must be congratulated massively. They could have gone into pointless jobs in the city printing money - they didn’t, they pursued new knowledge. Go them.

I think the NHS will cover it
Like PP my Mum was 12 years in a 24/7 nursing care home say £600,000 (not old age illness so covered by NHS)
Plus 12 years of NHS doctor neurologist/psychiatrist/GP care (say 6 times a year)
Plus years of allied NHS service e.g.physio/OT/dietician etc. (monthly)

Plus people with HD are working age so the person will be able to be economically active - working and paying taxes which I assume they factor in.

This is such amazing news. I hope that they offer the treatment to presymptomatic individuals and this disease becomes a disease of the past.