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First UK child to receive gene therapy for fatal genetic disorder is now healthy

livescience.com

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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#151
post #130

Earlier quoted context omitted.

Not sure I follow. It's not like a therapy with a sticker price of $3.8 million is more available in countries without nationalized healthcare.

Sure it is. Look at the Blue Cross Blue Shield plan for Michigan. It covers a number of gene therapies at a cost of $2.2M for one of them. Not all European countries pay for it. https://www.bcbsm.com/amslibs/content/dam/public/employers/d...

You can get private insurance in Europe too.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#152

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

The fact that there's a time=money=lives equivalency is kinda unintuitive at first glance, but when you think about it for a bit it will become obvious.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#153
post #135

We are truly living in a very interesting time. AI is already "smarter" than a big chunk of the population, Musk's company is expected to land human onto the mars soon, gene therapy is able to cure rare diseases that was unthinkable just a few decades ago, a certain type of colon cancer was 100% cured in a trial. Just shockingly amazing!

Yup! We're still fucked with climate change though. That one isn't fixable.

Depends on what you mean.

Geoengineering can stop global warming if necessary, but bringing back a destroyed ecosystem is effectively impossible.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#154
post #135

We are truly living in a very interesting time. AI is already "smarter" than a big chunk of the population, Musk's company is expected to land human onto the mars soon, gene therapy is able to cure rare diseases that was unthinkable just a few decades ago, a certain type of colon cancer was 100% cured in a trial. Just shockingly amazing!

> Musk's company is expected to land human onto the mars soon I wouldn't make any bets based on Musk's posturing about Mars.

Adjust for Elon Time.

In general if Elon says it'll be done in two years time you can be confident it will take at least 3 years. Average of 4, but possibly more than 10

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#155

> Teddi and her sister Nala, age 3, were both diagnosed with MLD in April 2022, according to the NHS. Unfortunately, Nala was not eligible for Libmeldy because she'd already developed symptoms of the disorder. Holy shit. My god, I'm just thinking of the parents. "Sorry, we can only save one of your kids." "You can't give Nala the treatment?" "Well, we could . We won't, though." I'm sure there are good reasons, but Je…

The treatment works on the child without symptoms because in most cases neurological affections are irreversible. Since the child had not developed them, the therapy would essentially fix the body before it caused them.

In the case of the child who already shows neurological symptoms, the treatment can help stop the progression of the disease, but most likely not reverse the already existing damage. However, there are already treatments approved to help stop the progression of the disease, including enzyme replacement therapy (provide the enzyme in charge of degrading the sustrate through the bloodstream every so often).

Some would argue that if treatments with similar outcomes already exist and are approved, it could be unethical to test this experimental treatment on a patient (I disagree).

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#156

How exactly do you edit a gene? Like, it is very, very small thing. How do you even measure it, let alone replace it?

Biochemistry is wild! Measuring is an absolutely fascinating procedure. The predominant method is called sequencing by synthesis. Illumina is the company that basically rules the market for this technology, they have some decent explanatory videos if you’re interested. The first steps are to isolate DNA, then break it into small bits, replicate those small pieces thousands of times, and then anchor these to a glass s…

Thank you very much for the detailed explanation.

This is the kind of content I visit this site for.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#157

Earlier quoted context omitted.

There is no free lunch but money is not a constraint. Physical resources are a hard constraint. Labour is a hard constraint. Money is not. Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply. It’s no more (or less) complicated than any of the thousands of other fiscal decisions a government makes every year. There’s a fall…

The real constraint is indeed what that money buys, e.g the aforementioned person hours. And if you print money and inflate then the price of goods rises. So sure, you might only pay a bit more for the treatment but now fruits are more expensive and your population gets slightly more unhealthy.

>> and inflate

>> >> Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#158
post #30

Earlier quoted context omitted.

In Europe they have these things called planes, it's like a tube you enter and sit for a few hours and once exit you are in a driving distance to American hospitals where you can pay and receive the same treatment as everyone else without nationalised healthcare. Best of the both worlds.

What's more, Europe also has these private hospitals which aren't connected to the national healthcare system and which will provide you with top level care if you are willing to pay, no travel to US necessary. Private health insurance ( gasp! ) is also a thing, should you want to partake in that system.

Europe consists of many countries, and one of them is more than happy to take 9% / 5% of your income in exchange for one quite crappy public health care system - think whenever you need to see a specialist, you have to pay with your own money anyway (or wait months, years to get an appointment). If you want private health insurance, you have to fund the super expensive good for nothing public one anyway.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#159

you can't just simply state that he's healthy without actually seeing his lifespan

The headline claims the child is healthy, but the article itself says

> Roughly six months out from treatment, "Teddi is a happy and healthy toddler showing no signs of the devastating disease she was born with," the NHS statement reads.

and later

> In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms; these patients were able to break down sulfatides at normal rates and showed typical patterns of motor development, for example. The benefit of the therapy seemed to last several years, but at this point, "it is not yet clear whether it will persist life-long, and extended follow-up is needed," the EMA noted.

Which is much more nuanced.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#160
post #127

> Teddi and her sister Nala, age 3, were both diagnosed with MLD in April 2022, according to the NHS. Unfortunately, Nala was not eligible for Libmeldy because she'd already developed symptoms of the disorder. Holy shit. My god, I'm just thinking of the parents. "Sorry, we can only save one of your kids." "You can't give Nala the treatment?" "Well, we could . We won't, though." I'm sure there are good reasons, but Je…

> In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms Does that mean clincal trials didn't show a benefit for patients who had already developed symptoms? Does it mean it wasn't studied in patients who had already developed symptoms? I don't think it's unreasonable that the NHS won't spend large amounts of other people's money on a treatment tha…

> Does that mean clincal trials didn't show a benefit for patients who had already developed symptoms? Does it mean it wasn't studied in patients who had already developed symptoms?

It likely wasn't studied.

It's a pretty new treatment, so the vendors start with clinical trials for use cases where the clearest/greatest impact is expected, and maybe later on move to related use cases.

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