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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.

#111

Earlier quoted context omitted.

This is what happens when healthcare is for profit.

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.

How is not weighing risk not for profit, when not weighing it is crucial for profit?

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

#112

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…

Most doctors are very bad at data as well. Few understand statistics. That does not help.

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

#113
post #108

Earlier quoted context omitted.

> Didn't inform the family that the treatment could possibly kill their daughter From the article: > 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. Qiu said getting the dose right was critical, but infusing the viruses directly into Mei’s spinal fluid, rather than the blood, wo…

Right, part of his whole pitch was that he had a new cutting edge gene therapy that was more precise than CRISPR. I think the parents were definitely obsessed with "fixing their daughter", but it seems like Qiu didn't try at all to "snap them out of it" by mentioning how the treatment he was attempting could also kill her.

Of course he didn't try to discourage them. He wanted to do this type of research/experimentation, which is almost certainly why he was known to the communities the parents were a part of (the article says the parents learned of him in a "WeChat group in which parents of children with autism and similar disorders trade advice").

This is a form of doctor shopping. If you really want something, you can find someone who is willing to provide it, even in fields like medicine where practitioners are expected to adhere to rigorous ethical standards.

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

#115
post #56

Earlier quoted context omitted.

I didn't read it as making the doctor a monster. What I saw in the story is an example of how the road to hell is paved with good intentions.

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 literally funding the creation, for the first time ever, of a highly complex treatment essentially custom-made for their daughter's condition. This required a lot of detailed and expensive science, with the generation of a mouse model of the disease, followed by development of the treatment, and then the testing of this in monkeys. In US/EU, this work would likely cost millions of dollars/euros, and it's not unreasonable that it would also cost a significant sum of money in China.

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

#116
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…

> May be such infusion would really serve as a vaccine prepping the immune system for the main infusion later?

This is correct and exactly the problem. You don't want the immune system to react to the virus, but giving it exposure to the virus will nearly guarantee a reaction. This is why they were checking that the patient didn't have antibodies already.

Here is an article discussing this issue: https://pmc.ncbi.nlm.nih.gov/articles/PMC10673641/

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

#118

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…

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 stop the cancer growth, 30% chance that it had no impact. I guess you have to take that chance when you have no other option. I don't blame the hospital (which was terrible) or the treatment, it was the last card left. And our loved ones end up in the 30% statistic and maybe as another anecdote for the risks section, but we know what we they went through and we won't forget.

It was tough because my dad was suffering, but he still was active and had at least a few months left without the treatment and I know that at that point in his battle he did this treatment to keep fighting for us rather than for him, even though it was he who had to suffer through it. So I am going to honor his memory and keep fighting until the end as well.

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

#119

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 tradeoffs would be regarded as malpractice if something went wrong.

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

#120

Earlier quoted context omitted.

This is what happens when healthcare is for profit.

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.

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