6 ms·

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

369 points by Shortness8 2mo ago

singpolyma3 2mo ago
Headline is the whole story. Sometimes a therapy doesn't work. Especially a new one
- ncallaway 2mo ago
  > Headline is the whole story. That's just not true. From the article: > According to official documents and accounts provided by the girl’s parents, the hospital had allowed Qiu’s experimental treatment to proceed under a regulatory provision that does not require approval from national regulators. After the child’s death, the hospital paid a modest fine to a local health authority but Qiu was not publicly sanctioned. That's not in the headline, but is an important part of the story. Also from the article: > Seven experts in fields including genetics, virology, and bioethics who reviewed details of the Nature study and the clinical trial for Science and Retraction Watch expressed concern that Qiu and his team downplayed the trial’s risks in describing them to the parents, overlooked safety signals in animal studies, and proceeded even though success was unlikely. Also not in the headline, but also an important part of the story. Finally: > The girl’s parents, who requested that Science use pseudonyms for them and their daughter for privacy reasons, have decided to tell her story now because they are angry about what they feel is a lack of accountability by the researchers and the institutions. “Learning the reality of these missing safeguards has fundamentally changed how we now view the entire project,” says the father, a software engineer. He asked that he be called Jason, his wife Linda, and their daughter Mei (Chinese for “beautiful”). “We did not realize how unusual and dangerous many of the arrangements were.” Also not in the headline, also an important part of the story.
  - dmix 2mo ago
    So it was a regulatory escape hatch for highly experimental medicine and it didn't work out? > The girl would be the first person in the world to receive a gene-editing therapy directed at the brain. The first person to ever try something comes with risks...
    - ncallaway 2mo ago
      And were those risks adequately communicated to the people taking them? According to the story, they weren't. That's a Big Fucking Deal, and is absolutely a significant part of the story. Was that in the headline?
      - dmix 2mo ago
        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 stated in the consent form: > The platelets in her blood also dropped to dangerous levels. It was the exact sequence of symptoms that the consent form had warned the family about. Qiu should have been more cautious in some of their communication with the obviously emotional parents, but this is pretty far from a scam and these aren't nobody doctors within China. He had a postdoc from UC San Diego, was a well known neuroscientist in China, published in Nature, etc.
        shunhe 2mo ago
        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
        dmix 2mo ago
        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.
  - cogman10 2mo ago
    Not to mention the part where it appears that the entire department decided to try and scam these parents. > Jeremy Sugarman, a medical doctor and bioethicist at Johns Hopkins University, says it’s not unusual for a family to bear the costs of developing a personalized treatment. But, according to text messages shared by Jason and Linda, Qiu also asked the couple to pay other members of the research team directly, through informal arrangements they found increasingly troubling. Qiu kept on adding on new back channel payments and seemed to keep ballooning the costs. I have to wonder if the procedure started because it was ready or because the parents ran out of resources.
    - neonstatic 2mo ago
      The part about gifts he received from the father in person is particularly vile.
      - rainprincess 2mo ago
        I think it's a part of the culture of guanxi (relations) in China.
        neonstatic 2mo ago
        Good point
- fabian2k 2mo ago
  It's worse than that. The therapy likely killed her, and her condition wasn't fatal in the first place.
  - processing 2mo ago
    "global developmental delay" yeah this is tragic - this kid didn't need to go through this.
  - asqueella 2mo ago
    I was curious what non-fatal condition would make the parents so desperate (to participate in a first-in-human trial): > Mei was diagnosed with global developmental delay .. some of Mei’s behaviors .. were associated with autism. > CHD3 mutations produce a condition called Snijders Blok-Campeau syndrome > people with the mutation often have a normal life expectancy, but their symptoms vary widely. Most have slightly larger than normal heads, and about two-thirds have intellectual deficits. Moderate to severe cases may be nonverbal, suffer from seizures and heart problems, and have fluid-filled voids in their heads.
- deleted 2mo ago
  [deleted]
- slashdave 2mo ago
  https://en.wikipedia.org/wiki/Hippocratic_Oath https://en.wikipedia.org/wiki/Hippocratic_Oath
- bryan_w 2mo ago
  This is the most suspicious comment I've ever read on this site
fabian2k 2mo ago
There's so many ethical problems with the events as described in the article. The worst to me seems to be that the researchers/doctors seem to have downplayed the risks here. Which for a never before tried gene therapy that is meant to work inside the brain are absolutely enormous. The ethical issues around the money seem minor in comparison with that and the fact that they seem to have ignored similar side effects in the monkey experiments.
- cowlby 2mo ago
  Reminds me of the TGN1412 drug trial where patients had severe immune reaction and almost died. Sad that the story suggests they ignored all the signs that would suggest humans would have a bad immune reaction to it and proceeded anyway. https://www.reddit.com/r/Documentaries/comments/jrraz7/when_a_drug_trial_goes_wrong_emergency_at_the/ https://www.reddit.com/r/Documentaries/comments/jrraz7/when_...
  - mft_ 2mo ago
    With TGN1412, it wasn't that the right work wasn't done, or that results ignored, but rather that the regulations in place at the time weren't right to deal with that sort of drug. Following what happened, the regulations were changed around the world to be more cautious and suitable.
- trollbridge 2mo ago
  I’m very puzzled why this wasn’t replicated in eg a study in rats first (where you duplicate the mutation, and then attempt to cure it).
  - austin-schick 2mo ago
    The article says that they did do this: > Qiu’s team had engineered mice to have a human version of the CHD3 gene with their daughter’s mutation, R1025W, which results in a protein with the amino acid tryptophan where there should be an arginine. The mutant pups developed autismlike traits and didn’t squeak as much as normal mice when separated from their mothers. When the researchers repaired that mutation, the pups developed normally.
deleted 2mo ago
[deleted]
oceansky 2mo ago
I can't possibly imagine what the parents are going through
- vintagedave 2mo ago
  I found the description of how they moved apartment, but kept the old one with her room, very saddening. I get it - an aversion or a shrine, hard to say, or both I suspect (in grief I have had both feelings at the same time), but I get it.
Terr_ 2mo ago
> The paper had an enthusiastic reception. “These promising results might pave the way for the development of an effective clinical treatment,” Kevin Bender, a neuroscientist at UC San Francisco, wrote in an accompanying commentary. At the time, Bender had no idea that a girl had received it and was already dead. Meanwhile, Chinese state media, CCTV, called the work “the first ray of hope” for “countless families suffering such diseases.” Oof, there's something deeply unjust about that, a kind of "adding insult to fatality." People celebrating that a new treatment will save children and give hope... with no acknowledgement that it was just tried and cost you both.
- drekipus 2mo ago
  "Millions of deaths don't matter if we can save just one." is an increasingly common take.
  - cucumber3732842 2mo ago
    If you really want people to fall for the like you put fractional life on one side of the equation and whole numbers of life on the other "millions of people ought to toil for untold millions of man hours totaling to hundreds of lifetimes to save a few lives" and all that.
  - Departed7405 2mo ago
    Source ?
    - drekipus 2mo ago
      Israel
      - throw098422 2mo ago
        There haven’t been “millions of deaths” in Israeli conflicts, even if you combine all sides since the start of its existence. You are just looking to derail yet another unrelated conversation into a Middle-East flamewar. This happens often on threads that are negative about China or Russia.
aa55b9c43e47364 2mo ago
[flagged]
nubg 2mo ago
> 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-wasting) called and how can we exterminate it?
- wrafawfwg 2mo ago
  All your comments boil down to: Why are people people and not machines?
- catlover76 2mo ago
  This kind of thing is pretty standard, and you accordingly come across as rather boorish. But it is generally referred to as "creative non-fiction", and I agree it can often be overdone.
- trollbridge 2mo ago
  This is good quality journalism; human-interest details like this are important.
- raincole 2mo ago
  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.
  - hyperpape 2mo ago
    Did you describe what happened in a neutral tone?
  - orangedog 2mo ago
    They're writing about a child that died, I don't think they hav any other motive other than the reader to feel sadness or outrage and given it is a dead child, they should not.
wnevets 2mo ago
> 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, for instance—were associated with autism.
- ToucanLoucan 2mo ago
  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.
  - slashdave 2mo ago
    It was a genetic disorder. Stop with the silly labeling.
  - orangedog 2mo ago
    That's not what happened here. The team was too ambitious and not careful enough but they were not trying to kill the child. I just don't think there is any reason to put that additional context into what happened here.
    - ryandrake 2mo ago
      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.
      - sokoloff 2mo ago
        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”.
tliltocatl 2mo ago
TL;DR: - The girl had a really rare genetic disorder, traceable to a single-base mutation, that result in intellectual disability. Her case was quite mild, she was verbal and only had a learning disability, other cases are often far more severe. - They went for using adeno-associated virus as vector to deliver a CRISPR payload. It caused kidney and liver damage (AFAIUK due to immune response, not the virus itself? So hard to predict from an animal model.) which turned out fatal. - The family paid a significant share of the research funding and some off-record financial favors to the research team. - The research team's recent publication in Nature didn't mention the case at all (they basically chose keep silent about failures). The whole story has quite some Flowers for Algernon vibes except real life is way more cynical and sad. And I disliked the book back when I read it because it felt like a weepie just for the sake of weeping.
lconnell962 2mo ago
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 complicated parts of a human body after all. Ethics and safty concerns seemed to get ignored all the way to the actual treatment speaks like the money and potential fame was all the medical staff involved were concerned with. The parent's aren't blameless, they wanted to fix a child who probably would have lived with a below average quality of life. The article claims they were mislead to believe this treatment was safer than it actually was. The treatment vehicle seems like the cause of death as reported toward the end, but there are words about the animal testing that imply this could have been a known risk with the treatment as a whole. The number of things being found with hindsight remind me of "move fast and break things" development. It really sounds like cutting edge (bio)tech has ethics get ignored when money and fame are on the table.
- throwway120385 2mo ago
  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.
  - ndiddy 2mo ago
    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 stripped of any mention of the failed human trial and of any negative side effects caused by the immune system reacting to the virus The most generous interpretation of this is that Qiu and his team were racing to be the first to cure a mental disorder using gene editing and neglected to take the necessary safety precautions, but that still makes him a horrible person because it resulted in the unnecessary death of a child just because he was hoping to be first. I'm glad the parents decided to go public about this (especially since they must feel devastated about their daughter's death) if it means he won't get to try the same risky procedure on other children.
arjie 2mo ago
What? The monkeys all had problems! Why do this? Just do PGT for your other kids. Come on, dude! Non lethal condition. Deranged behaviour. We did IVF with PGT and these days they tell you about carrier screening super early and everything here in California. I wouldn’t experiment on a real-life living human like this. A last ditch effort to save a life perhaps but come on, dude. If you're interested in the actual process of PGT and IVF, I wrote it down here: https://wiki.roshangeorge.dev/w/IVF https://wiki.roshangeorge.dev/w/IVF Well-trodden ground and quite safe.
- arjie 2mo ago
  I told a friend of mine this and he pointed me to an article by another friend of ours: https://www.chinatalk.media/p/a-cancer-patients-tour-of-chinese https://www.chinatalk.media/p/a-cancer-patients-tour-of-chin... Here's the key quote: > The tradeoff is pretty straightforward: the US system emphasizes uniform standards and upfront rigor, while China’s [investigator-initiated trial] model pushes decision-making closer to the doctor and the patient, making it easier to start trials quickly and iterate as data comes in It's clear that they are prioritizing iteration over standardization - which is a good pathway to exploration but will yield results like this. In the end, we're all going to benefit from the new research coming out of China as they subject themselves to this high-variance policy.
Hansenq 2mo ago
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.
- laughing_man 2mo ago
  China officially ended its one-child policy in 2015. Barring medical reasons, there was no reason for their daughter to be their only child.
  - Our_Benefactors 2mo ago
    It’s been ended officially but due to social pressures to “put all your resources into one child” as the parents were accustomed to, having just one child is still a strong cultural factor. The CCP can’t just flip a switch and suddenly the whole culture aligns with having many children.
    - Departed7405 2mo ago
      In fact, China fertility rare went down no lower than 1.5 during the one-child policy. In 2024 however, it was 1.03 child/woman, and last year it sank to less than 1 child per woman. But of course there are huge disparities between cities and rural areas.
moralestapia 2mo ago
"The young girl tugged on her mother’s hand as they pressed through the doors ..." Zack-D films tier writing there, disgusting.
HedonicEscal8r 2mo ago
There are many children with life-threatening rare diseases that would be much better candidates for risky experimental treatment like this. Very sad case.
- array4277 2mo ago
  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.
trhway 2mo ago
"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 immune system for the main infusion later? - though immune system killing the viruses may be nullifying the treatment - then may be when doing such therapy the patient needs to be [somewhat] immunosuppressed similar to transplant situations?
- fwip 2mo ago
  The article mentions that she was given an immunosuppressant (prednisone), but that a more comprehensive course of immunouppressants could have been given.
- elzbardico 2mo ago
  Acquired immunity is complicated territory. Doing this way could make a future adverse reaction more certain. But I don't think you're far from the correct procedure. Clinical trials are structured in phases. And AFAIK, safety is the first thing to be determined.
- slashdave 2mo ago
  It could negate the treatment (the vector will be neutralized by the immune system).
  - trhway 2mo ago
    that is why after thinking a bit i edited the post adding the opposite track of thought - immunosuppression.
- 395trek 2mo ago
  that's why in clinical trials you first do phase 1 human subjects where they are healthy and take small sub-therapeutic doses to test safety/tolerability. once it's established safe in humans they do phase 2 where they test if it works on sick people. Yeah I know this is different with transduction but they could at least test the vector first, maybe they did? Maybe some kind of lentivirus vector
maxall4 2mo ago
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, but infusing them directly into someone's brain and expecting nothing bad to happen is, in my view, crazy.
- timy2shoes 2mo ago
  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.
  - arjie 2mo ago
    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.
- gus_massa 2mo ago
  IANAMD. I remember something about that virus in the brain are super bad, and there is an additional protection to avoid virus and random substances entering the brain. They injected the virus in the medula, that is inside the protection membrane. In an ideal case, is it a good idea?
- fgimenez 2mo ago
  [Note that I work in this field and have co-founded a CNS AAV company] This isn't the correct takeaway. AAV are one of the most complex drug modalities and carry considerable risk when used incorrectly. This story is tragic and violates pretty much every ethical consideration for a clinician researcher. Especially ones that are treating children of desperate parents. That said, AAV are one of the most powerful delivery mechanism we have to deliver gene therapies to the brain. Uniqure has shown the first efficiacious treatment of Huntington's disease with intraparenchymal delivery of AAV5, Zolgensma is a brain targeted AAV9 to treat SMA, Kebilidi is an intraparenchymal AAV2 that treats AADC deficiency. The general approach should be to keep dose as low as possible and minimally expose the periphery. AAV9 at large doses delivered intrathecally without standardized immunosuppression is simply insane.
deleted 2mo ago
[deleted]
shunhe 2mo ago
This shows how important disclosures are. A field like this cannot learn if unsuccessful human experiments disappear while the corresponding animal work is published as promising.
senderista 2mo ago
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).
- api 2mo ago
  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."
  - alightsoul 2mo ago
    This is what happens when healthcare is for profit.
    - deleted 2mo ago
      [deleted]
    - m0llusk 2mo ago
      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.
Scroll_Swe 2mo ago
[flagged]
morkalork 2mo ago
Eugenics wars when?
Shitty-kitty 2mo ago
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.
- jwrallie 2mo ago
  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.
amoorthy 2mo ago
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 physiotherapist. What's particularly galling is the recovery from back surgery would've taken at least 6-9 months of rehab and my father's PSP was already sapping his motor skills and yet the surgeon was pushing ahead. Even with my mom I've had to intervene on several occasions against the doctor's utterly idiotic ideas. Thank goodness for AI to at least make us conversant to ask the right questions of these doctors.
- uurrnn 2mo ago
  Physios really are great
  - harry8 2mo ago
    Can be. They vary like _every_ other profession.
1saadcodes 2mo ago
Cases like this are heartbreaking, but they're also a reminder that failures like this ones need to be published just as prominently as success. Gene editing is still a young field, and if negative outcomes remain hidden, other researchers can't properly assess risks or make improvements
- Departed7405 2mo ago
  I am baffled by Nature saying would this have been communicated during peer-review this would have been taken into consideration, but because this was discovered after it doesn't count anymore. It's like a teacher saying if you get caught cheating during the exam you will be punished, but if it's after you can get "scott-free."
blondie9x 2mo ago
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?
- HedonicEscal8r 2mo ago
  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/ 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_syndrome https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_... https://www.nature.com/articles/s41586-026-10113-6 https://www.nature.com/articles/s41586-026-10113-6
raver1975 2mo ago
I hope they get their money back
Simulacra 2mo ago
[flagged]
RS-232 2mo ago
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.
throwaway89864 2mo ago
Here are some illustration of people living with this disease: https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_syndrome https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_...
deleted 2mo ago
[deleted]
ro_bit 2mo ago
If families paying for experimental treatment is illegal in China, how did these researchers get away with it?
tsoukase 2mo ago
In the West medical procedures and therapies remain under the most conservative restrictions, which on one side saves lives due to potential failures, on the other it slows progression and innovation. We keep error type I extremely low. It seems China doesn't hold such a tradition and is unjustifiably daring. I don't doubt how COVID escaped.
htlemur_bobby 2mo ago
Honestly we can’t hate them for trying to id rather be dead than live with that disease.

369 points by Shortness8 2mo ago

singpolyma3 2mo ago

Headline is the whole story. Sometimes a therapy doesn't work. Especially a new one

ncallaway 2mo ago
> Headline is the whole story. That's just not true. From the article: > According to official documents and accounts provided by the girl’s parents, the hospital had allowed Qiu’s experimental treatment to proceed under a regulatory provision that does not require approval from national regulators. After the child’s death, the hospital paid a modest fine to a local health authority but Qiu was not publicly sanctioned. That's not in the headline, but is an important part of the story. Also from the article: > Seven experts in fields including genetics, virology, and bioethics who reviewed details of the Nature study and the clinical trial for Science and Retraction Watch expressed concern that Qiu and his team downplayed the trial’s risks in describing them to the parents, overlooked safety signals in animal studies, and proceeded even though success was unlikely. Also not in the headline, but also an important part of the story. Finally: > The girl’s parents, who requested that Science use pseudonyms for them and their daughter for privacy reasons, have decided to tell her story now because they are angry about what they feel is a lack of accountability by the researchers and the institutions. “Learning the reality of these missing safeguards has fundamentally changed how we now view the entire project,” says the father, a software engineer. He asked that he be called Jason, his wife Linda, and their daughter Mei (Chinese for “beautiful”). “We did not realize how unusual and dangerous many of the arrangements were.” Also not in the headline, also an important part of the story.
- dmix 2mo ago
  So it was a regulatory escape hatch for highly experimental medicine and it didn't work out? > The girl would be the first person in the world to receive a gene-editing therapy directed at the brain. The first person to ever try something comes with risks...
  - ncallaway 2mo ago
    And were those risks adequately communicated to the people taking them? According to the story, they weren't. That's a Big Fucking Deal, and is absolutely a significant part of the story. Was that in the headline?
    - dmix 2mo ago
      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 stated in the consent form: > The platelets in her blood also dropped to dangerous levels. It was the exact sequence of symptoms that the consent form had warned the family about. Qiu should have been more cautious in some of their communication with the obviously emotional parents, but this is pretty far from a scam and these aren't nobody doctors within China. He had a postdoc from UC San Diego, was a well known neuroscientist in China, published in Nature, etc.
      - shunhe 2mo ago
        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
        dmix 2mo ago
        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.
- cogman10 2mo ago
  Not to mention the part where it appears that the entire department decided to try and scam these parents. > Jeremy Sugarman, a medical doctor and bioethicist at Johns Hopkins University, says it’s not unusual for a family to bear the costs of developing a personalized treatment. But, according to text messages shared by Jason and Linda, Qiu also asked the couple to pay other members of the research team directly, through informal arrangements they found increasingly troubling. Qiu kept on adding on new back channel payments and seemed to keep ballooning the costs. I have to wonder if the procedure started because it was ready or because the parents ran out of resources.
  - neonstatic 2mo ago
    The part about gifts he received from the father in person is particularly vile.
    - rainprincess 2mo ago
      I think it's a part of the culture of guanxi (relations) in China.
      - neonstatic 2mo ago
        Good point
fabian2k 2mo ago
It's worse than that. The therapy likely killed her, and her condition wasn't fatal in the first place.
- processing 2mo ago
  "global developmental delay" yeah this is tragic - this kid didn't need to go through this.
- asqueella 2mo ago
  I was curious what non-fatal condition would make the parents so desperate (to participate in a first-in-human trial): > Mei was diagnosed with global developmental delay .. some of Mei’s behaviors .. were associated with autism. > CHD3 mutations produce a condition called Snijders Blok-Campeau syndrome > people with the mutation often have a normal life expectancy, but their symptoms vary widely. Most have slightly larger than normal heads, and about two-thirds have intellectual deficits. Moderate to severe cases may be nonverbal, suffer from seizures and heart problems, and have fluid-filled voids in their heads.
deleted 2mo ago
[deleted]
slashdave 2mo ago
https://en.wikipedia.org/wiki/Hippocratic_Oath https://en.wikipedia.org/wiki/Hippocratic_Oath
bryan_w 2mo ago
This is the most suspicious comment I've ever read on this site

fabian2k 2mo ago

There's so many ethical problems with the events as described in the article. The worst to me seems to be that the researchers/doctors seem to have downplayed the risks here. Which for a never before tried gene therapy that is meant to work inside the brain are absolutely enormous. The ethical issues around the money seem minor in comparison with that and the fact that they seem to have ignored similar side effects in the monkey experiments.

cowlby 2mo ago
Reminds me of the TGN1412 drug trial where patients had severe immune reaction and almost died. Sad that the story suggests they ignored all the signs that would suggest humans would have a bad immune reaction to it and proceeded anyway. https://www.reddit.com/r/Documentaries/comments/jrraz7/when_a_drug_trial_goes_wrong_emergency_at_the/ https://www.reddit.com/r/Documentaries/comments/jrraz7/when_...
- mft_ 2mo ago
  With TGN1412, it wasn't that the right work wasn't done, or that results ignored, but rather that the regulations in place at the time weren't right to deal with that sort of drug. Following what happened, the regulations were changed around the world to be more cautious and suitable.
trollbridge 2mo ago
I’m very puzzled why this wasn’t replicated in eg a study in rats first (where you duplicate the mutation, and then attempt to cure it).
- austin-schick 2mo ago
  The article says that they did do this: > Qiu’s team had engineered mice to have a human version of the CHD3 gene with their daughter’s mutation, R1025W, which results in a protein with the amino acid tryptophan where there should be an arginine. The mutant pups developed autismlike traits and didn’t squeak as much as normal mice when separated from their mothers. When the researchers repaired that mutation, the pups developed normally.

deleted 2mo ago

[deleted]

oceansky 2mo ago

I can't possibly imagine what the parents are going through

vintagedave 2mo ago
I found the description of how they moved apartment, but kept the old one with her room, very saddening. I get it - an aversion or a shrine, hard to say, or both I suspect (in grief I have had both feelings at the same time), but I get it.

Terr_ 2mo ago

> The paper had an enthusiastic reception. “These promising results might pave the way for the development of an effective clinical treatment,” Kevin Bender, a neuroscientist at UC San Francisco, wrote in an accompanying commentary. At the time, Bender had no idea that a girl had received it and was already dead. Meanwhile, Chinese state media, CCTV, called the work “the first ray of hope” for “countless families suffering such diseases.” Oof, there's something deeply unjust about that, a kind of "adding insult to fatality." People celebrating that a new treatment will save children and give hope... with no acknowledgement that it was just tried and cost you both.

drekipus 2mo ago
"Millions of deaths don't matter if we can save just one." is an increasingly common take.
- cucumber3732842 2mo ago
  If you really want people to fall for the like you put fractional life on one side of the equation and whole numbers of life on the other "millions of people ought to toil for untold millions of man hours totaling to hundreds of lifetimes to save a few lives" and all that.
- Departed7405 2mo ago
  Source ?
  - drekipus 2mo ago
    Israel
    - throw098422 2mo ago
      There haven’t been “millions of deaths” in Israeli conflicts, even if you combine all sides since the start of its existence. You are just looking to derail yet another unrelated conversation into a Middle-East flamewar. This happens often on threads that are negative about China or Russia.

aa55b9c43e47364 2mo ago

[flagged]

nubg 2mo ago

> 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-wasting) called and how can we exterminate it?

wrafawfwg 2mo ago
All your comments boil down to: Why are people people and not machines?
catlover76 2mo ago
This kind of thing is pretty standard, and you accordingly come across as rather boorish. But it is generally referred to as "creative non-fiction", and I agree it can often be overdone.
trollbridge 2mo ago
This is good quality journalism; human-interest details like this are important.
raincole 2mo ago
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.
- hyperpape 2mo ago
  Did you describe what happened in a neutral tone?
- orangedog 2mo ago
  They're writing about a child that died, I don't think they hav any other motive other than the reader to feel sadness or outrage and given it is a dead child, they should not.

wnevets 2mo ago

> 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, for instance—were associated with autism.

ToucanLoucan 2mo ago
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.
- slashdave 2mo ago
  It was a genetic disorder. Stop with the silly labeling.
- orangedog 2mo ago
  That's not what happened here. The team was too ambitious and not careful enough but they were not trying to kill the child. I just don't think there is any reason to put that additional context into what happened here.
  - ryandrake 2mo ago
    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.
    - sokoloff 2mo ago
      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”.

tliltocatl 2mo ago

TL;DR: - The girl had a really rare genetic disorder, traceable to a single-base mutation, that result in intellectual disability. Her case was quite mild, she was verbal and only had a learning disability, other cases are often far more severe. - They went for using adeno-associated virus as vector to deliver a CRISPR payload. It caused kidney and liver damage (AFAIUK due to immune response, not the virus itself? So hard to predict from an animal model.) which turned out fatal. - The family paid a significant share of the research funding and some off-record financial favors to the research team. - The research team's recent publication in Nature didn't mention the case at all (they basically chose keep silent about failures). The whole story has quite some Flowers for Algernon vibes except real life is way more cynical and sad. And I disliked the book back when I read it because it felt like a weepie just for the sake of weeping.

lconnell962 2mo ago

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 complicated parts of a human body after all. Ethics and safty concerns seemed to get ignored all the way to the actual treatment speaks like the money and potential fame was all the medical staff involved were concerned with. The parent's aren't blameless, they wanted to fix a child who probably would have lived with a below average quality of life. The article claims they were mislead to believe this treatment was safer than it actually was. The treatment vehicle seems like the cause of death as reported toward the end, but there are words about the animal testing that imply this could have been a known risk with the treatment as a whole. The number of things being found with hindsight remind me of "move fast and break things" development. It really sounds like cutting edge (bio)tech has ethics get ignored when money and fame are on the table.

throwway120385 2mo ago
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.
- ndiddy 2mo ago
  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 stripped of any mention of the failed human trial and of any negative side effects caused by the immune system reacting to the virus The most generous interpretation of this is that Qiu and his team were racing to be the first to cure a mental disorder using gene editing and neglected to take the necessary safety precautions, but that still makes him a horrible person because it resulted in the unnecessary death of a child just because he was hoping to be first. I'm glad the parents decided to go public about this (especially since they must feel devastated about their daughter's death) if it means he won't get to try the same risky procedure on other children.

arjie 2mo ago

What? The monkeys all had problems! Why do this? Just do PGT for your other kids. Come on, dude! Non lethal condition. Deranged behaviour. We did IVF with PGT and these days they tell you about carrier screening super early and everything here in California. I wouldn’t experiment on a real-life living human like this. A last ditch effort to save a life perhaps but come on, dude. If you're interested in the actual process of PGT and IVF, I wrote it down here: https://wiki.roshangeorge.dev/w/IVF https://wiki.roshangeorge.dev/w/IVF Well-trodden ground and quite safe.

arjie 2mo ago
I told a friend of mine this and he pointed me to an article by another friend of ours: https://www.chinatalk.media/p/a-cancer-patients-tour-of-chinese https://www.chinatalk.media/p/a-cancer-patients-tour-of-chin... Here's the key quote: > The tradeoff is pretty straightforward: the US system emphasizes uniform standards and upfront rigor, while China’s [investigator-initiated trial] model pushes decision-making closer to the doctor and the patient, making it easier to start trials quickly and iterate as data comes in It's clear that they are prioritizing iteration over standardization - which is a good pathway to exploration but will yield results like this. In the end, we're all going to benefit from the new research coming out of China as they subject themselves to this high-variance policy.

Hansenq 2mo ago

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.

laughing_man 2mo ago
China officially ended its one-child policy in 2015. Barring medical reasons, there was no reason for their daughter to be their only child.
- Our_Benefactors 2mo ago
  It’s been ended officially but due to social pressures to “put all your resources into one child” as the parents were accustomed to, having just one child is still a strong cultural factor. The CCP can’t just flip a switch and suddenly the whole culture aligns with having many children.
  - Departed7405 2mo ago
    In fact, China fertility rare went down no lower than 1.5 during the one-child policy. In 2024 however, it was 1.03 child/woman, and last year it sank to less than 1 child per woman. But of course there are huge disparities between cities and rural areas.

moralestapia 2mo ago

"The young girl tugged on her mother’s hand as they pressed through the doors ..." Zack-D films tier writing there, disgusting.

HedonicEscal8r 2mo ago

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

array4277 2mo ago
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.

trhway 2mo ago

"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 immune system for the main infusion later? - though immune system killing the viruses may be nullifying the treatment - then may be when doing such therapy the patient needs to be [somewhat] immunosuppressed similar to transplant situations?

fwip 2mo ago
The article mentions that she was given an immunosuppressant (prednisone), but that a more comprehensive course of immunouppressants could have been given.
elzbardico 2mo ago
Acquired immunity is complicated territory. Doing this way could make a future adverse reaction more certain. But I don't think you're far from the correct procedure. Clinical trials are structured in phases. And AFAIK, safety is the first thing to be determined.
slashdave 2mo ago
It could negate the treatment (the vector will be neutralized by the immune system).
- trhway 2mo ago
  that is why after thinking a bit i edited the post adding the opposite track of thought - immunosuppression.
395trek 2mo ago
that's why in clinical trials you first do phase 1 human subjects where they are healthy and take small sub-therapeutic doses to test safety/tolerability. once it's established safe in humans they do phase 2 where they test if it works on sick people. Yeah I know this is different with transduction but they could at least test the vector first, maybe they did? Maybe some kind of lentivirus vector

maxall4 2mo ago

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, but infusing them directly into someone's brain and expecting nothing bad to happen is, in my view, crazy.

timy2shoes 2mo ago
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.
- arjie 2mo ago
  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.
gus_massa 2mo ago
IANAMD. I remember something about that virus in the brain are super bad, and there is an additional protection to avoid virus and random substances entering the brain. They injected the virus in the medula, that is inside the protection membrane. In an ideal case, is it a good idea?
fgimenez 2mo ago
[Note that I work in this field and have co-founded a CNS AAV company] This isn't the correct takeaway. AAV are one of the most complex drug modalities and carry considerable risk when used incorrectly. This story is tragic and violates pretty much every ethical consideration for a clinician researcher. Especially ones that are treating children of desperate parents. That said, AAV are one of the most powerful delivery mechanism we have to deliver gene therapies to the brain. Uniqure has shown the first efficiacious treatment of Huntington's disease with intraparenchymal delivery of AAV5, Zolgensma is a brain targeted AAV9 to treat SMA, Kebilidi is an intraparenchymal AAV2 that treats AADC deficiency. The general approach should be to keep dose as low as possible and minimally expose the periphery. AAV9 at large doses delivered intrathecally without standardized immunosuppression is simply insane.

deleted 2mo ago

[deleted]

shunhe 2mo ago

This shows how important disclosures are. A field like this cannot learn if unsuccessful human experiments disappear while the corresponding animal work is published as promising.

senderista 2mo ago

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).

api 2mo ago
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."
- alightsoul 2mo ago
  This is what happens when healthcare is for profit.
  - deleted 2mo ago
    [deleted]
  - m0llusk 2mo ago
    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.

Scroll_Swe 2mo ago

[flagged]

morkalork 2mo ago

Eugenics wars when?

Shitty-kitty 2mo ago

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.

jwrallie 2mo ago
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.

amoorthy 2mo ago

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 physiotherapist. What's particularly galling is the recovery from back surgery would've taken at least 6-9 months of rehab and my father's PSP was already sapping his motor skills and yet the surgeon was pushing ahead. Even with my mom I've had to intervene on several occasions against the doctor's utterly idiotic ideas. Thank goodness for AI to at least make us conversant to ask the right questions of these doctors.

uurrnn 2mo ago
Physios really are great
- harry8 2mo ago
  Can be. They vary like _every_ other profession.

1saadcodes 2mo ago

Cases like this are heartbreaking, but they're also a reminder that failures like this ones need to be published just as prominently as success. Gene editing is still a young field, and if negative outcomes remain hidden, other researchers can't properly assess risks or make improvements

Departed7405 2mo ago
I am baffled by Nature saying would this have been communicated during peer-review this would have been taken into consideration, but because this was discovered after it doesn't count anymore. It's like a teacher saying if you get caught cheating during the exam you will be punished, but if it's after you can get "scott-free."

blondie9x 2mo ago

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?

HedonicEscal8r 2mo ago
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/ 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_syndrome https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_... https://www.nature.com/articles/s41586-026-10113-6 https://www.nature.com/articles/s41586-026-10113-6

raver1975 2mo ago

I hope they get their money back

Simulacra 2mo ago

[flagged]

RS-232 2mo ago

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.

throwaway89864 2mo ago

Here are some illustration of people living with this disease: https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_syndrome https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_...

deleted 2mo ago

[deleted]

ro_bit 2mo ago

If families paying for experimental treatment is illegal in China, how did these researchers get away with it?

tsoukase 2mo ago

In the West medical procedures and therapies remain under the most conservative restrictions, which on one side saves lives due to potential failures, on the other it slows progression and innovation. We keep error type I extremely low. It seems China doesn't hold such a tradition and is unjustifiably daring. I don't doubt how COVID escaped.

htlemur_bobby 2mo ago

Honestly we can’t hate them for trying to id rather be dead than live with that disease.