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

131–140 of 248 posts

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

#131

Post by laughing_man »

Hansenq wrote: post #47

This is a tragic story. But it is also a story about the lengths that parents in China will go to improve the quality of life for their only child or to "save face" to their social circle about how their child is performing relative to others. It's quite tragic that they felt the need to lean into this treatment and quite tragic that they were led on. Just a sad story all around.

China officially ended its one-child policy in 2015. Barring medical reasons, there was no reason for their daughter to be their only child.

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

#132

Post by MSKJ »

decimalenough wrote: post #124
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…

If he had succeeded, though, he would have been a hero, both to the parents and the world. And the same racing that we are condemning as monstrous would have saved other children as well. Obviously I'm not saying he is a hero or that what he did is right, just pointing out that the ethical tradeoffs in medical research like this get very complicated very fast. Ultimately it boils down to the trolley problem: is sacri…

It doesn't seem like success was of particular import to the doctor, rather the goal seems to have been to get a 'test subject'

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

#133

Post by Supermancho »

caraffle wrote: post #93
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 is fear mongering at best and unbelievable. Why would a surgeon know anything about anesthesia risk? Anesthesia regurarly cancels cases due to safety concerns - there's no "protocol" or "getting in trouble" (what does that even mean), that's standard of care. "Anesthesia" is a doctor too and ultimately holds the call on if they will put a patient under or not.

This is matches my experience, having been under at least 30 times.

The phrase:

"Why would a surgeon know anything about anesthesia risk?"

could be misleading to someone who has never undergone (or just didn't understand) what a pre-surgery anesthesiologist consult is for.

Surgeries are performed by teams. Anesthesia is one aspect of the procedure and most of the doctors know some, while surgeons know a great deal. The anesthesiologist's job, as a physician, is to monitor, adjust, and assess risk during the procedure. This disclosure, if it happened, was during the consultation.

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

#134

Post by RS-232 »

Surely this was preventable? I am surprised that immunologists and immunosuppressants weren’t part of the protocol here. Injecting trillions of viruses into spinal fluid sounds like a recipe for triggering a cytokine storm.

It’s a tragic outcome, but her death is not in vain. The results need to be published for the public benefit, advancement in this area of research, and preventing similar outcomes.

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

#135

Post by Supermancho »

amatecha wrote: post #123
caraffle wrote: post #93

Earlier quoted context omitted.

This is fear mongering at best and unbelievable. Why would a surgeon know anything about anesthesia risk? Anesthesia regurarly cancels cases due to safety concerns - there's no "protocol" or "getting in trouble" (what does that even mean), that's standard of care. "Anesthesia" is a doctor too and ultimately holds the call on if they will put a patient under or not.

> Why would a surgeon know anything about anesthesia risk? Idunno, maybe because their entire career is performing surgeries which include anaesthesia and they have worked for potentially decades alongside anaesthesiologists? For instance, I've worked in the same type of role (programmer) for my entire career and I somehow know a ton about the disciplines adjacent or closely related to mine, especially because I work…

Graciously, I think this was the point they were trying to make, with an inappropriately sarcastic intro.

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

#136

Post by solenoid0937 »

amatecha wrote: post #123
caraffle wrote: post #93

Earlier quoted context omitted.

This is fear mongering at best and unbelievable. Why would a surgeon know anything about anesthesia risk? Anesthesia regurarly cancels cases due to safety concerns - there's no "protocol" or "getting in trouble" (what does that even mean), that's standard of care. "Anesthesia" is a doctor too and ultimately holds the call on if they will put a patient under or not.

> Why would a surgeon know anything about anesthesia risk? Idunno, maybe because their entire career is performing surgeries which include anaesthesia and they have worked for potentially decades alongside anaesthesiologists? For instance, I've worked in the same type of role (programmer) for my entire career and I somehow know a ton about the disciplines adjacent or closely related to mine, especially because I work…

Medicine is not programming.

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

#137

Post by vintagedave »

raincole wrote: post #28
nubg wrote: post #17

> The young girl tugged on her mother’s hand as they pressed through the doors of the hospital in Shanghai. She was 6 years old, bouncing along in a pink jacket and blue pants decorated with cartoon bears. Behind them, her father rolled a large suitcase with everything the child needed for the weeklong stay: stuffed animals, Play-Doh, an iPad loaded with episodes of Peppa Pig. What's this style of "journalism" (time-…

Treat those proses as warnings. They mean the writers have decided to guide your emotions in certain direction instead of portraying what happened in a neutral tone.

I’ve written articles in this style. The goal is to be evocative of the story. Simple factual accounts carry less weight than the reader being present in the situation. To remain truthful and accurate you describe factually; it is not counter to that to also describe in a way that situates, evokes, humanizes something that is about the experience.

This is about two uncertain, trusting parents taking their young daughter for treatment. It is in every way one of the most frightening human experiences. In that light, this is a light, gentle touch by the author trying to communicate to you the reader the humanity behind the facts.

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

#138

Post by NoLinkToMe »

esotericrunner wrote: post #118
diskzero wrote: post #96

Earlier quoted context omitted.

That is terrible and I am glad someone broke ranks to inform you of the risk. My wife had a very agressive triple-hit lymphoma and CAR-T was eventually suggested. Fortunately, the medical team was very honest about how hard it impacts your body, with very likely chance of death. My wife decided to do it, and it almost killed her. The team actually had to "shut down" the process, so she didn't benefit completely from…

Sorry for your loss, I feel your pain. Your wife was very brave to give it a shot and take the fight to the limit, and you should remember her courage and honor her memory and keep fighting till the end regardless of what hardships you encounter in life. The treatment actually killed my dad. I may be wrong but it appears with the treatment there was a 30% chance of some recovery, 40% chance that it would at least sto…

Thank you both for sharing.

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

#139

Post by Eisenstein »

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…

> 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 be applied to it. The basic fact is that it should be some form public service and not reliant on a system which has as its incentive maximizing profits, and attempting to regulate it into being patient first and not profit first will always have side-effects like you pointed out.

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

#140

Post by HedonicEscal8r »

blondie9x wrote: post #104

Does anyone know what was the actual illness the girl had? I see a T was supposed to be a C but what is that? What are the long term implications?

She had Snijders Blok-Campeau syndrome, which is caused by mutations in the CHD3 protein, which manages the structure of DNA. In her case, it was this missense variant: https://www.ncbi.nlm.nih.gov/clinvar/variation/422607/

The severity of Snijders Blok-Campeau syndrome can vary, but it sounds like Mei only suffered from mild intellectual disability.

https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_... https://www.nature.com/articles/s41586-026-10113-6

Post reply on HN