Hacker News Board Hacker News, browsed like a classic message board
Live data from Hacker News

Couple pay >$800k for a gene-editing therapy for their daughter. She died.

science.org

Post reply on HN
Print view View original

221–230 of 248 posts

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#221

Post by altairprime »

mft_ wrote: post #209
altairprime wrote: post #171

Earlier quoted context omitted.

Her distinctiveness has been added the collective. That's what experimental medicine is for! So if you approach this story from an amoral "ethics-free" standpoint (which is easy for me but, thankfully, difficult for most) their money has technically benefited human science, however problematic the circumstances. I have some serious glee at seeing the quantity of moral output being generated in this discussion. HN's c…

> So if you approach this story from an amoral "ethics-free" standpoint (which is easy for me but, thankfully, difficult for most) their money has technically benefited human science, however problematic the circumstances. I'd argue it's marginal. For some sort of scientific benefit, at least, new knowledge (e.g. what to do or what not to do) is needed. * For the experts involved, the approach , while novel in histor…

Yes, it's absolutely marginal. It's 'single datapoint' at the absolute best. I'm not arguing for doing more of this. I'm just pointing out that, in the true spirit of (early) Walter Bishop, better shoddy-one than data-none if moral quandaries are of no concern.

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#222

Post by qsera »

kashunstva wrote: post #207
qsera wrote: post #201

Earlier quoted context omitted.

Quite frankly, no one expects that and that is not where the disappointment comes from. The disappointment comes from the fact that doctors are mostly clueless and follow checklists, and thus prescribes measures without having a deep understanding (or interest even) of the situation. Why? Because these people (doctors) were never curious to begin with. 99% of the never cared to understand biology before they started…

> 99% of the never cared to understand biology before they started Medical school. They are only in for the $$$ and status This is a very cynical take on the subject and I can’t imagine it’s backed by any data. As a retired medical doctor, my observation differs; idealism and curiosity appear to me far more common among medical trainees than you give credit. I spent my entire career in teaching hospitals. I do despis…

>This is a very cynical take on the subject and I can’t imagine it’s backed by any data.

Imagine if being a doctor did not pay as well. Imagine it only pays just enough to sustain a small family. What percentage of your trainees do you think would remain in the course, for the love of the trade?

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#223

Post by refurb »

fgimenez wrote: post #183
maxall4 wrote: post #58

I'm honestly quite shocked that the physicians/scientists involved would choose to use an AAV for a brain-targeted gene therapy. There is just so much data demonstrating that these vectors are quite immunoreactive: most of the approved gene therapies based on AAVs carry black box labels for liver failure caused by an immune reaction to the viral capsid. Admittedly, AAVs are the most derisked vector for gene therapies…

[Note that I work in this field and have co-founded a CNS AAV company] This isn't the correct takeaway. AAV are one of the most complex drug modalities and carry considerable risk when used incorrectly. This story is tragic and violates pretty much every ethical consideration for a clinician researcher. Especially ones that are treating children of desperate parents. That said, AAV are one of the most powerful delive…

Zolgensma acts on the peripheral motor neurons not the CNS

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#224

Post by jmalicki »

qsera wrote: post #222
kashunstva wrote: post #207

Earlier quoted context omitted.

> 99% of the never cared to understand biology before they started Medical school. They are only in for the $$$ and status This is a very cynical take on the subject and I can’t imagine it’s backed by any data. As a retired medical doctor, my observation differs; idealism and curiosity appear to me far more common among medical trainees than you give credit. I spent my entire career in teaching hospitals. I do despis…

>This is a very cynical take on the subject and I can’t imagine it’s backed by any data. Imagine if being a doctor did not pay as well. Imagine it only pays just enough to sustain a small family. What percentage of your trainees do you think would remain in the course, for the love of the trade?

So you're talking about doctors in Europe.

Europe has plenty of doctors.

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#225

Post by LoveMortuus »

senderista wrote: post #76

Many years ago I was attending pre-surgery for a hip replacement surgery for my sister, who had known severe reactions to anesthesia (actually required a tracheotomy for a previous reaction). The anesthesiologist asked to speak to us privately and informed us that in their opinion, my sister had maybe a 1/3 chance of not surviving the surgery. They also mentioned that this was a breach of protocol and they could get…

Off topic, but:

>will refuse to give you the slightest hint of any number attached to risk (I know, I've tried many times).

reminds me of my lawyer... (I lost)

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#226

Post by iso1631 »

modeless wrote: post #150
Eisenstein wrote: post #139

Earlier quoted context omitted.

> Love to hear all the time how the "free market" is ruining healthcare. Of course everyone knows a "free market" is when you have profit caps, supply restrictions (residency cap), government-run marketplaces, byzantine tax subsidies tying insurance to employment for no reason, etc etc What people are talking about with regard to health insurance being ruined by being market based is that the profit motive should not…

Removing the "evil" profit motive has always been an argument for socialism. If it would work for healthcare, why not everything?

Well it does work for healthcare pretty much everywhere.

Why is the fire service socialised in the US. Why are roads. Why is water?

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#227

Post by iso1631 »

alightsoul wrote: post #82
api wrote: post #78

Earlier quoted context omitted.

US healthcare is very strange in that on one hand it pinches pennies and is incentivized to not fund treatment, but on the other hand there seems to be some kind of internal incentivize to spend liberally and do everything. The whole system is a mess of perverse incentives. I've often described it as "everything bad about socialized medicine combined with everything bad about privatized medicine."

This is what happens when healthcare is for profit.

The UK has healthcare for profit, and not just the private healthcare options like BUPA -- GP surgeries are privately owned.

Does it happen there?

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#228

Post by iso1631 »

laughing_man wrote: post #120
m0llusk wrote: post #106

Earlier quoted context omitted.

It is more complicated than that. Markets have the structure and regulations we give them. Health care gets set up and delivered with very different concepts and guidance in various places regardless of whether it is for profit.

We don't have a true free-market in health care, so regulations and various programs are meant to create an environment where we get the benefits of a free market while still having universal availability and a universal "standard of care". Of course it doesn't work very well. That's asking too much of any system.

the only problem with communism is nobody's actually tried #TrueCommumismTM

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#229

Post by XorNot »

sokoloff wrote: post #85
XorNot wrote: post #75

Earlier quoted context omitted.

I don't know where you're getting that number from. For all dental anesthesia the risk rate seems to be literally 1 in a million [1] and the various law firms which popped up during that search suggested better then 1 in 300,000 at least. Which also omits the base level mortality of dental complications wisdom teeth are likely to cause. [1] https://pmc.ncbi.nlm.nih.gov/articles/PMC5535496/

Table 1 in your link does not support 1 in a million for general anesthesia procedures, unless I’m badly misreading it. Nor does the discussion section nor overall summary figure, which is 3 in a million for all forms of anesthesia (local, sedation, and general), where you have to at least exclude local anesthesia (the most common type) as having a fundamentally lower risk of death than sedation or GA.

You're right I misread it - the headline number is inline with the other quoted values of closer to 1 in 320,000 or so.

Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.

#230

Post by red-iron-pine »

nickff wrote: post #149
thruway515 wrote: post #147

Earlier quoted context omitted.

No it's not like any other field. There's no such thing as a 10x doctor. A '10x' doctor is just as likely to kill you as any other especially when it comes to experimental never before done procedure. In fact a 10x doctor like Qiu is more likely to take risks. This is medicine not a software shop where we measure productivity by number of commits or number of surgeries per hour except by 10x you mean 10times more lik…

What exactly do you mean by "10x doctor"? Some surgeons have a 10x higher 'revision rate' than others, and from what I understand, that may be an understatement. Many of the statistics which would indicate the ones to avoid are not tracked, precisely because the doctors do not want them tracked.

doctor stats are also misleading af, since the busiest times often require the best, most reliable docs, and they also have the highest rates of fatalities

7pm-2am on friday is when all of the nasty cases come in, and the best surgeon in the world ain't gonna save many of them.

Post reply on HN