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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.

#171

Post by altairprime »

mft_ wrote: post #115
ndiddy wrote: post #56

Earlier quoted context omitted.

He's pretty clearly a monster or at least monster adjacent. He and his team * Took hundreds of thousands of dollars under the table to develop the treatment * Neglected to take steps that would determine whether the treatment was safe to use on humans * Didn't inform the family that the treatment could possibly kill their daughter * After the treatment did kill their daughter, he published the study anyway except str…

> * Took hundreds of thousands of dollars under the table to develop the treatment I'm in two minds about this. On the one hand, at face value the funding and payments seem dubious - especially with the mention of one of the doctors returning his payments, suggesting they were for his personal enrichment, rather than payment for the costs of the treatment development and trial. On the other hand, the parents were lit…

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 culture is largely Bay Area tech, which has also been a key innovation driver in ethics-free amorality such as Meta and Theranos. May our own reflection in this medical science funhouse mirror encourage us to express such intense moral and ethical concern at not only our tech social media site HN, but more importantly at our often-amoral tech workplaces as well.

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

#172

Post by antisthenes »

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…

> If there are 10x and 0.1x engineers, you bet there are 10x and 0.1x doctors.

So if we already have a massive doctor shortage, including 0.1x doctors, you can begin to guess how awful the medical system is in the US.

You could be paying exorbitant rates only to end up with a 0.1x doctor, because there was literally no one else within 30 miles in a rural area.

Pretty depressing to think about.

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

#173

Post by solumunus »

thruway515 wrote: post #156
nickff wrote: post #149

Earlier quoted context omitted.

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'

Obviously they simply mean highly talented compared to their peers.

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

#174

Post by Analemma_ »

antisthenes wrote: post #172
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…

> If there are 10x and 0.1x engineers, you bet there are 10x and 0.1x doctors. So if we already have a massive doctor shortage, including 0.1x doctors, you can begin to guess how awful the medical system is in the US. You could be paying exorbitant rates only to end up with a 0.1x doctor, because there was literally no one else within 30 miles in a rural area. Pretty depressing to think about.

It’s one reason why I will absolutely not move to a rural area, no matter what the benefits are to cost-of-living and getting away from the city. Rural America is getting completely emptied of all its medical providers, because it’s impossible to provide medical services at a profit. Doubly-so if you need any kind of specialist care; in rural areas, forget about it.

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

#175

Post by array4277 »

HedonicEscal8r wrote: post #51

There are many children with life-threatening rare diseases that would be much better candidates for risky experimental treatment like this. Very sad case.

This is what makes this whole thing so unethical. All the researchers cared about was that the parents said they had a lot of money and didn't care to spend it.

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

#176

Post by dmix »

shunhe wrote: post #69
dmix wrote: post #34

Earlier quoted context omitted.

This is pretty silly. If you're paying $860k for something (note this is USD being spent within China), you can afford to do basic research on what you're getting involved with, which they apparently did: > The parents had heard about serious side effects, including deaths, caused by other gene therapies, and knew the greatest risk would be Mei’s immune response to the massive dose of virus. The risk was explicitly s…

There is a meaningful difference between knowing that gene therapy can cause serious complications in general and being told that every treated monkey developed moderate to severe liver damage, with one also showing kidney damage consistent with the mechanism that ultimately killed the child. Informed consent was pretty hard to get here

The main issue is the hospital ethics committee signed off on a private clinical trial based on early positive results, but before the final toxicology report was finished... the whole program in China is about commercially funded research (meaning it didn't go under the usual Chinese FDA-type review which is more thorough). This family was privately paying for studies on mice and giving the doctor hundreds of thousands of dollars to explore this avenue. It also says he did share the toxicology report with family beforehand and they still went ahead with it anyway. This is the report showing a monkey died of liver failure, the same thing that ended up killing their daughter.

I believe it was a very dumb idea to use their kid in something very experimental, especially in China. But lets not pretend the family didn't play a huge role, with many chances to have it double checked with other scientists before putting their daughters life at risk. They 100% had the means to do so but chose to gamble.

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

#178

Post by kazinator »

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…

1/3rd chance of not surviving is like suddenly dashing across a busy freeway without looking, from a crouched position behind a bush. That's what it means to send the person into that. Yikes!

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

#179

Post by brianwawok »

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…

What do they call the guy that got the lowest grade in medical school? Doctor

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

#180

Post by arjie »

timy2shoes wrote: post #152
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…

These are all fair complaints, but DB-OTO works the same way for the otoferlin-related hearing loss: it's packaged on two viral AAV vectors. It's given to much smaller babies, though there are some Chinese reports of a similar gene therapy in teens. So it's clearly not just the dual-vector but also the target etc.
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