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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.

#71

Post by slashdave »

ToucanLoucan wrote: post #23
wnevets wrote: post #18

> When Mei was 4, one of her kindergarten teachers pulled Linda aside: Mei didn’t draw or write as well as the other kids and her language skills weren’t developing normally. Her mother might want to get her evaluated, the teacher said. In March 2023, Mei was diagnosed with global developmental delay, a broad label with many causes. Specialists explained that some of Mei’s behaviors—the funny sounds she liked to make…

It says so much about how our society treats the neurodivergent that it's considered a non-extreme opinion that a dead child is preferable to an autistic one.

It was a genetic disorder. Stop with the silly labeling.

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

#72

Post by slashdave »

trhway wrote: post #57

"Seven days after the girl’s medical team infused trillions of viruses carrying the recipe for the base editor into her spinal fluid, she died of a severe immune reaction linked to the therapy" questions of a layman - couldn't they initially do a small infusion of the [may be even weakened version of that] viruses to check for the immune reaction? May be such infusion would really serve as a vaccine prepping the immu…

It could negate the treatment (the vector will be neutralized by the immune system).

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

#74

Post by [deleted] »

lconnell962 wrote: post #31

An article about the complicated issue that is a child with a non-lethal developmental disorder getting a treatment that ends with the tragedy of the headline. The article might be sensationalizing the situation, but it makes the doctor out like a monster and as the facts read I can't say I disagree. That the treatment was inconclusive in the animal studies isn't particularly shocking. The brain is one of the more co…

[deleted]

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

#75

Post by XorNot »

sokoloff wrote: post #55
ryandrake wrote: post #43

Earlier quoted context omitted.

I think what OP was trying to ask was why would a parent be willing to put their child at lethal risk, using an experimental treatment, for a condition that itself is not lethal? I don't have a kid with autism or a learning disability so I don't know how I'd handle it, but if I did, I'd probably choose to live with it and help the child make the best of it. I would not be reaching for someone's research project.

Parents put their children at lethal risk (albeit small) for non-lethal conditions all the time. Wisdom teeth removal under general anesthesia is around 1 in 100,000. It doesn’t sound so crazy if the risks were presented as “only 10x as risky as wisdom teeth extraction”.

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/

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

#76

Post by senderista »

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 in trouble for talking to us directly, but their conscience wouldn't let them do otherwise. We returned and asked the surgeon if they really thought the risk justified any potential benefit. The surgeon shrugged and said "probably not, feel free to call it off". 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.

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).

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

#78

Post by api »

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…

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."

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

#79

Post by trhway »

slashdave wrote: post #72
trhway wrote: post #57

"Seven days after the girl’s medical team infused trillions of viruses carrying the recipe for the base editor into her spinal fluid, she died of a severe immune reaction linked to the therapy" questions of a layman - couldn't they initially do a small infusion of the [may be even weakened version of that] viruses to check for the immune reaction? May be such infusion would really serve as a vaccine prepping the immu…

It could negate the treatment (the vector will be neutralized by the immune system).

that is why after thinking a bit i edited the post adding the opposite track of thought - immunosuppression.

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

#80

Post by PearlRiver »

taurath wrote: post #49
lconnell962 wrote: post #31

An article about the complicated issue that is a child with a non-lethal developmental disorder getting a treatment that ends with the tragedy of the headline. The article might be sensationalizing the situation, but it makes the doctor out like a monster and as the facts read I can't say I disagree. That the treatment was inconclusive in the animal studies isn't particularly shocking. The brain is one of the more co…

> The number of things being found with hindsight remind me of "move fast and break things" development. It is the same mentality in all of business rn, everyone is ruthless and doesn’t give a single fuck about consequences because they won’t be coming for another 2 years at least and people have a short memory

Also if you have a few hundred million bucks you will still have your mansion if you get it wrong.
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