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

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

#151

Post by ButlerianJihad »

itishappy wrote: post #107
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…

> The lesson for me is that you must advocate for yourself and your loved ones in the medical system, because doctors will not do it for you; they may not even perform the most basic risk assessments. And you have to try to quantify risk yourself, because doctors will refuse to give you the slightest hint of any number attached to risk (I know, I've tried many times). Doctors vary wildly, and that's part of the issue…

https://youtu.be/6h6dhevlb0w?is=t-cLAwe5vBuKx_fZ

Your Coworker Who's a 10× Engineer

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

#152

Post by timy2shoes »

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…

Another compounding issue is that they had to package the vector into two parts, which then have to both infect the same cell to get any effect. Which means you have to at least double the dose to get similar coverage compared to a single AAV vector (and actually more than double). Seems like a easy recipe for liver toxicity. Which is why most companies doing AAV therapy either target the liver or the eye (where AAV doesn't escape to the liver).

I feel like the parents were not well enough informed of the risks, and the PI rushed the therapy to be famous. Not the first time this has happened, and not the last, sadly.

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

#153

Post by jwrallie »

Shitty-kitty wrote: post #92

It is important to note that in China, developmental delays are treated with ridicule for the affected and as a source of shame for the families. I'm not passing judgment on the parents, I'm just pointing out that how society treats developmental delays is extremely important to the quality of life of these people. Here in the US autism was considered a "mental illness" until the 1980's.

I think this is an important nuance that may be missed in the article. The teacher pulling the parent aside to ask her to check her child made me remember what my partner said about the time they moved to China as a kid and how they were treated in school because they were falling behind their peers. (In that case because they couldn’t keep up with the natives due to language skills.)

I too do not mean this particular teacher is to blame rather than the way society works.

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

#154

Post by [deleted] »

genericresponse wrote: post #119
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…

This seems very unusual on many, many levels. I hate to ask, but were you a child when this happened? If so, are you sure you were in all the conversations. First- There are less than 600 total hip replacements conducted each year on children in the United States. Second- It would be unusual for this surgery to be even recommended without something serious being addressed. Third- Failure to discuss risks and tradeoff…

[deleted]

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

#155

Post by itishappy »

thruway515 wrote: post #147
itishappy wrote: post #107

Earlier quoted context omitted.

> The lesson for me is that you must advocate for yourself and your loved ones in the medical system, because doctors will not do it for you; they may not even perform the most basic risk assessments. And you have to try to quantify risk yourself, because doctors will refuse to give you the slightest hint of any number attached to risk (I know, I've tried many times). Doctors vary wildly, and that's part of the issue…

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…

Yeah, I don't mean it literally, nor do I necessarily agree with the concept of 10x engineers in general, but it's a useful shorthand. I would bet doctors vary somewhat less than other fields, but my family has been surprised and burnt by the variance still there, and I'm the only non-doctor!

On the topic of 10x surgeries per hour, the 1800s were an interesting time! This guy in particular:

https://en.wikipedia.org/wiki/Robert_Liston

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

#156

Post by thruway515 »

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.

You'll have to ask the OP what they meant by 10x. I assume they meant it in the programmer/hacker sense of producing more work in the same amount of time than their peers or being a 'rockstar'

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

#157

Post by uurrnn »

amoorthy wrote: post #98

Two years ago, my then 81-yr old father, who had Progressive Supranuclear Palsy, was recommended to get back surgery to relieve chronic and debilitating pain. The neurosurgeon said it's routine surgery and he's done it on people much older. We ultimately decided against it and found that a simple wedge pillow to sleep on relieved his pain. A bloody $50 pillow. And this came from a recommendation from a physiotherapis…

Physios really are great

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

#158

Post by foco_tubi »

thruway515 wrote: post #147
itishappy wrote: post #107

Earlier quoted context omitted.

> The lesson for me is that you must advocate for yourself and your loved ones in the medical system, because doctors will not do it for you; they may not even perform the most basic risk assessments. And you have to try to quantify risk yourself, because doctors will refuse to give you the slightest hint of any number attached to risk (I know, I've tried many times). Doctors vary wildly, and that's part of the issue…

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…

The point is, there are excellent doctors, good doctors, bad doctors, and awful doctors who should probably have their licensure revoked; just like engineers, or any other profession, there is a spectrum to the quality of service/talent/care.

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

#159

Post by qotgalaxy »

modeless wrote: post #105
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."

Some people thought it was a good idea to cap [administration cost + profit] of health insurance companies as a percentage of premiums. As a result, health insurance administrators have two ways to increase their compensation: they can compete to acquire more customers, or they can artificially inflate costs per customer. Guess which one is easier! Now you don't have to wonder why medication is half the cost when you…

[dead]

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

#160

Post by tomcam »

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…

> Keep in mind that nobody on the care team had previously discussed any risk or indeed any tradeoffs whatsoever. This was at one of the best-regarded children's hospitals in the USA.

I bet you live in the Seattle area. Similar experience with my daughter.

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