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Couple pay >$800k for a gene-editing therapy for their daughter. She died.
(science.org)
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by Shortness8
by Shortness8
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.
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.
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).
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.
https://www.reddit.com/r/Documentaries/comments/jrraz7/when_...
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.
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.
I too do not mean this particular teacher is to blame rather than the way society works.
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."
https://en.wikipedia.org/wiki/Snijders_Blok%E2%80%93Campeau_...
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.
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.
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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
Well-trodden ground and quite safe.
- 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.
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.
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
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?
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Zack-D films tier writing there, disgusting.
What's this style of "journalism" (time-wasting) called and how can we exterminate it?
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.
Doctors vary wildly, and that's part of the issue. For instance, my oncologist had zero problem rattling off mortality statistics. I've personally had doctors try to sell me surgery before identifying the issue, but I've also had doctors successfully talk me out of what would have been useful procedures by offering their risk/benefit assessment unprompted.
It's like any other field. If there are 10x and 0.1x engineers, you bet there are 10x and 0.1x doctors. A 1hr intake appointment isn't anywhere near enough time to judge even for folks in the field.
But sadly, with doctors, someone who provides 10x value to the patient might not be the same ones that provide 10x value to the Hospitals that employ them. Which means real 10x doctors might be less and less visible.
At least in software, you don't need a certificate or association with an institution to practice the trade. So you can be self employeed while free to deliver 10x value to your customers..
The doctors obviously also talked to the patient and he or she obviously felt that just reducing the pain was worth the risk (because a replacement hip will still be long-term painful, so it's an improvement, not a solution. This will have been discussed extensively with the patient) ...
Or the patient may even have felt that the risk was in fact also a solution to the pain. The patient may have chosen not to talk about this to their relatives.
When you're really young medicine can feel like it solves problems permanently, especially when you're still growing. This is almost never actually the case. Medicine mitigates, makes bad situations livable for longer, restores part of your previous health. If you treat it like a solution once you're ... say 50 ... you will be severely disappointed and that can lead to drastic decisions. Or if you live your life like some people do "medicine will fix it, let's JUMP" you're likely to be disappointed, suddenly.
The disappointment comes from the fact that doctors are mostly clueless and follow checklists, and thus prescribes measures without having a deep understanding (or interest even) of the situation.
Why? Because these people (doctors) were never curious to begin with. 99% of the never cared to understand biology before they started Medical school. They are only in for the $$$ and status. And I will never trust a doctor, or even a programmer who is not curious about the specific situation or the problem that I am having. And such people are becoming rarer and rarer to find...
This is a false equivalency, as the 10x engineer is measured differently than a great doctor. A 10x doctor in your analogy should mean "this doctor is least likely to kill you during surgery", or "is better at following checklists".
The "best" doctors I've met have off the charts systems analysis skills, and they were in all kinds of specializations. Human physiology can be quite counter intuitive at times, and great doctors have great observability skills.
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.
Your Coworker Who's a 10× Engineer
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I have zero idea if what the doctor is telling me is accurate or not. I'm not a doctor.
This is why people are moving away from main stream medicine, in my opinion.
I had a rare and very complicated surgery for my cancer. I researched it heavily, and going into the appointment I already knew how the surgery should be performed. I asked the surgeon many questions, including how often he performs this surgery, for how many years, and how many complications he has. He was a bit offended. I also asked him how he would do the surgery, and he told me a rarer, more complicated method that is typically reserved for pre-chemo patients (chemo melts your insides, makes some surgery more complicated).
I told him this was unusual, not standard of care, and that I was skeptical. And he methodically laid out why I was a good candidate even though I’ve had chemotherapy. The surgery was very successful, and he was a fantastic surgeon.
But you still have to push. And if you don’t get the answers you need, you go somewhere else.
My dad enjoys seeing if the chatbots can diagnose odd stuff and they do very well to his great delight.
I will warn you, though, that if you’re a poor user of LLMs you will get bad results so don’t do this if you can’t prompt any frontier chatbot appropriately.
Someone shared the BRAIN acronym for making decisions.
Can't remember it exactly, but it was like ask / think about benefits, risks, alternatives, intuition, doing Nothing (for now).
I think the Nothing one was the most important, what happens if I don't take action and defer the decision. Medical professionals often make you feel like a decision is urgent when sometimes it is not.
_how_ do you advocate? If I could avoid delegating to experts, I wouldn’t be in the situation in the first place.
What other type of medicine is there? Most "non-mainstream" things are pseudoscience nonsense.
I bet you live in the Seattle area. Similar experience with my daughter.
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 the treatment. The suffering was pretty immense as well. She died a few months later regardless.
We are (were) both engineers and we did wonder how many might get streamrolled by the whole medical process, where you do get this odd combination of extreme indifference and optimistic exuberance.
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.
Penicillin, antibiotics, and many vaccines have only come into general use within the last 80 years which is what one or two lifetimes. Many/every medical procedure starts somewhere everyone just hopes they don’t start with them.
You might as well tie them to a post and shoot an apple off their head with a rifle from 50 yards.
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."
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
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>will refuse to give you the slightest hint of any number attached to risk (I know, I've tried many times).
reminds me of my lawyer... (I lost)
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.
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 with them every day.
Fear mongering bullshit.
For reference, this happened about 20 years ago.
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* 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.
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, would minimize the threat of a reaction because it would bypass the kidneys and liver.
The article paints a picture of well-educated parents who were incredibly focused and dedicated to their "mission", which was basically to "cure" their daughter's condition using gene editing, something that had never been done before. They participated in groups, recorded the conversations with the doctor and ostensibly did enough research to have concerns in the first place.
The doctor violated every ethics rule in the book and should never be allowed to touch another human being, but I don't think it's fair to pretend that the parents were unwitting.
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
The fact that the parents had to make further efforts to go through their daughters death to warn others just feels wrong.
Wouldn't be the first time. Nor the first time the actual evidence of effectiveness of the treatment (i.e. even when working as intended) was ignored.
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> 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.
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Getting in your car has risk, so does riding a bike. They shouldn’t be coin flips.
I just don't think there is any reason to put that additional context into what happened here.
They did not set out to kill the child. And the autism wasn't even the biggest issue. The issue is the developmental delays that were expected to compound the more the brain was left untreated.
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The far-more common sentiment that I see among anti-vaxxers is that the risk of death from childhood diseases like chicken pox are either zero or close-to-it, and that the risk of receiving autism from the vaccine is at least one-in-ten. These numbers aren't true, of course, but in their minds, they're weighing a negligible-chance of terrible outcome against a moderate chance of bad outcome.
I'm sure there's a few extremists of course, who would say they prefer a dead child to an autistic one (same as how some parents feel about gay kids), but it does not represent most of the people in these movements.
> 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.
> 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...
That's a Big Fucking Deal, and is absolutely a significant part of the story.
Was that in the headline?
> 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.
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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.
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/
That's a problem for some types of RNA/DNA vaccines where they use a virus as a vector. You can usually only use a specific kind of virus once per patient.
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.
Reading articles posted on HN is optional as are most things on the internet. If you don’t like the style in the first paragraph, stop reading. Getting fewer eyeballs on an article is typically a way to “exterminate” a style. Be aware, the articles that make the biggest waves tend to be long form feature writing. Given their influence and popularity you might find your extermination efforts to be quixotic.
Is this literally true "bouncing along in a pink jacket and blue pants decorated with cartoon bears"?
or just what the author thought the scene could have looked like?
On top of that Science is a reputable publication. They have fact checkers on staff. For a sensitive article like this it would have been independently gone over with a fine tooth comb. The fact checker will have seen a photo, or spoken directly with the family. This is all standard stuff in high level journalism like this.
There are journalism 101 courses you can audit to learn more about features, fact checking and how all this works. It seems like it would go a long way in improving your knowledge. Things like accusing journalists of fabricating things is a VERY serious accusation in that world. The writer is not going to make up the lede in a Science article on something that could be easily refuted by the parents and ruin their entire career.
It’s ok to not like feature writing or be ignorant of its existence and how it gets made. Calling for extermination and characterizing it as hallucinated slop with no evidence out of that ignorance is strangely aggressive.
I used Opus 4.8 to avoid Fable guardrail hair-trigger, but you can use ChatGPT as well.
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I looked up what neutral tone is, and it seems to be more about how you write than what you write. https://pressbooks.ccconline.org/medianewsandreporting/chapt...
This feels weird to me. It's like a newspaper describing a mass-killing without interviewing survivors because they would have emotions.
And also, neutrality is a myth, especially in news reporting. What you can do is using unloaded language and be balanced between parties.But being neutral doesn't mean you don't have values. Preferring live over death, and justice/honesty over injustice/cruelty.
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.
But it is generally referred to as "creative non-fiction", and I agree it can often be overdone.
But of course there are huge disparities between cities and rural areas.
This is a very cynical take on the subject and I can’t imagine it’s backed by any data. As a retired medical doctor, my observation differs; idealism and curiosity appear to me far more common among medical trainees than you give credit. I spent my entire career in teaching hospitals. I do despise the ostentatious display of amassed wealth among some doctors; but wouldn’t take a general message about the profession from that.
Imagine if being a doctor did not pay as well. Imagine it only pays just enough to sustain a small family. What percentage of your trainees do you think would remain in the course, for the love of the trade?
Europe has plenty of doctors.
Trying medicine because "you've heard that ..." is a fancy way to commit suicide, or worse. And yes, a great deal of people want to do that, until of course, they have to face the consequences. Then they want it fixed, except there is no fix. No doctor should want to be part of that, and these Chinese "researchers" should be made to answer for manslaughter for what they did.
Surely the story we're talking about illustrates this problem? For all we know the gene-editing worked! That was NOT the problem that killed the girl. Congrats, your "curiosity-based treatment" would have made the girl 10% smarter in 10 years. Probably. Unfortunately, she's dead in less than a week. Congratulations!
Is that REALLY something you'd like to hear about anyone you know? Would that satisfy your curiosity? Big risks, even ones your not aware of, are NOT a reasonable price for trying out medical treatments.
(At least that's how I've heard it phrased before.)
On the topic of 10x surgeries per hour, the 1800s were an interesting time! This guy in particular:
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.
7pm-2am on friday is when all of the nasty cases come in, and the best surgeon in the world ain't gonna save many of them.
You can ask for specific ways this can happen. But that would be point less. Human behavior and incentives work in ways that are beyond specifics.
Everyone here probably knows this already, but forgets it when it comes to medical domain.
For example, I have very weird/non standard genetics when it comes to processing medications, so I've had to learn which classes of drugs I need very small doses of or that I can't take at all. (Some drug classes aren't made in small enough doses for me to take safely). So I have to relay this to doctors and we make our medication decisions accordingly.
Or if you run hot or cold. My body temperature is usually a ~ 96.5, so I'm feeling awful by the time my temp hits 99.5, even though that doesn't register as a problem generally.
Or how I pushed to get a mammogram covered before it is ordinarily because my mother, grandmother, and great-grandmother have all had 3+ rounds of triple-negative breast cancer even though they don't have the BRCA genes: the standard recommendation doesn't fit our family and I am high risk despite not having the standard markers for being so.
Do extensive research before hand. AI can aide research, but you have to prompt it right. Tell it to do web searches, and tell it to only use trusted vetted sources and papers.
And the approach here isn’t to take the research at face value. It’s to take the research and derive questions, and then ask your doctor. You can be aggressive.
I had a rare and very complicated surgery for my cancer. I researched it heavily, and going into the appointment I already knew how the surgery should be performed. I asked the surgeon many questions, including how often he performs this surgery, for how many years, and how many complications he has. He was a bit offended. I also asked him how he would do the surgery, and he told me a rarer, more complicated method that is typically reserved for pre-chemo patients (chemo melts your insides, makes some surgery more complicated).
I told him this was unusual, not standard of care, and that I was skeptical. And he methodically laid out why I was a good candidate even though I’ve had chemotherapy. The surgery was very successful, and he was a fantastic surgeon.
But you still have to push. And if you don’t get the answers you need, you go somewhere else.
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.
The people who need it most are often the least profitable to treat and the least able to pay for it. That's because being sick negatively impacts your earning potential. The sicker you are the less you can afford a way to pay for treatment to not be sick. It's analogous to the problem with debtors' prison.
Obscure or low-survival-rate conditions are almost by definition unprofitable to treat. Rare condition? Stage four cancer? It's most profitable to let you suffer or die. There's not enough of a market for the former and the latter has a low probability of leaving someone behind to pay the bill.
Old age care can't be profitably offered on insurance or credit. Old? Pay cash or die.
Demand is not infinite because (excluding certain neuroses) people generally do not want health care. They only get it when they need it. Demand does not skyrocket with reduced price or free availability, so the system doesn't automatically just drown in demand when you take the cost away.
Competition is sparse because talent is scarce and the cost means there tend to be only one or a few hospitals covering an area. Specialty services and general practitioners are different, but for advanced and expensive care it tends to be not quite a natural monopoly but almost one. It's like how there's not very many space launch companies. Because it's so f'ing hard the supply of competitors is small.
Lastly, because customers are ignorant (very few people are qualified to evaluate health care tech or treatments), quackery is structurally more profitable than medicine that works. It's hard to have a healthy market when fraud is massively easier and more profitable and the customer finds it hard to tell the difference. Add to that the fact that try before you buy and comparison shopping are almost impossible, especially for serious medical care. You can't exactly toss out or return a surgery.
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Does it happen there?
Of course it doesn't work very well. That's asking too much of any system.
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.
> serious side effects, including deaths, caused by other gene therapies
That sounds like they were informed of risks for other therapies, not this one.
And TBH that framing is still suspect: if someone told me that the greatest risk was immune response and we have a way to minimize it, I'd take that to mean it's safe. That's not the right conclusion, but that's what most people would get from the phrasing. This is "how to lie with statistics". Maybe they were told something else though.
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.
I'd argue it's marginal. For some sort of scientific benefit, at least, new knowledge (e.g. what to do or what not to do) is needed.
* For the experts involved, the approach, while novel in historical terms, was application of known techniques. Further, to prove the treatment, they'd have needed a positive outcome, which sadly wasn't the case. So, some technical benefit here, but hindered by the negative outcome (and the possible retraction of the paper).
* They haven't really learned much more about what not to do, as some of the major criticisms of the study were that because of the approach taken and (arguably) the corners cut, it was predictably dangerous. Again, all of this was known - it was the failure to apply established principles that was the problem.
* It appears that (apart from this article) no details of the exact case have been released. A thorough write-up of the science and the clinical study would possibly add some benefit to science, but that's not been done.
Also, I am pretty sure this wouldn't have been allowed in the EU and the US, at least not with that few studies on animals. And with good reasons.
Also, you forget that the company probably also payed a lot for this, so it isn't like the total costs is limited to what was illegally paid to the researchers.
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 sacrificing a few worth saving many others?
A lot of institutional review and peer review is in place to prevent people from making mistakes like believing that they have a powerful, low-risk gene editing technique and that they can simply apply it at will. The most generous interpretation is that Qiu really thought he could do it, and that even if the side effects happened that he would be able to control them and save the daughter.
> monster
> monster
Perhaps if his genes were properly modified, then he could become nearly human in the court of public opinion.
>But nowhere did the form explicitly indicate that any of this could end in death, nor did that come up during any conversations with Qiu or the other doctors, Jason and Linda say.
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”.
Which also omits the base level mortality of dental complications wisdom teeth are likely to cause.
That's why we need external controls, ethical commites and so on.
I have multiple sclerosis and at the time of my diagnosis the best treatment available to me in terms of slowing the progression of MS had a risk of triggering a rare brain infection that would kill you. So it was a choice between treatments that didn't work as well but had lower risk and treatments with greater odds of being able to walk/work/not be in horrifying constant pain in 10 years but also a small chance of death.
These decisions can end up being horribly complicated, and they are definitely revealing in terms of your values.
> 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.
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.
Why is the fire service socialised in the US. Why are roads. Why is water?
Nor does the discussion section nor overall summary figure, which is 3 in a million for all forms of anesthesia (local, sedation, and general), where you have to at least exclude local anesthesia (the most common type) as having a fundamentally lower risk of death than sedation or GA.