There are many children with life-threatening rare diseases that would be much better candidates for risky experimental treatment like this. Very sad case.
Couple pay >$800k for a gene-editing therapy for their daughter. She died.
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Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#52> When Mei was 4, one of her kindergarten teachers pulled Linda aside: Mei didn’t draw or write as well as the other kids and her language skills weren’t developing normally. Her mother might want to get her evaluated, the teacher said. In March 2023, Mei was diagnosed with global developmental delay, a broad label with many causes. Specialists explained that some of Mei’s behaviors—the funny sounds she liked to make…
It says so much about how our society treats the neurodivergent that it's considered a non-extreme opinion that a dead child is preferable to an autistic one.
This seems to be a rather extreme takeaway..?
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#53An article about the complicated issue that is a child with a non-lethal developmental disorder getting a treatment that ends with the tragedy of the headline. The article might be sensationalizing the situation, but it makes the doctor out like a monster and as the facts read I can't say I disagree. That the treatment was inconclusive in the animal studies isn't particularly shocking. The brain is one of the more co…
Wealth can override pretty much anything; ethics, decency, common sense. This is a lesson humans have had thousands of years and countless examples to learn from and still haven't managed.
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#54> When Mei was 4, one of her kindergarten teachers pulled Linda aside: Mei didn’t draw or write as well as the other kids and her language skills weren’t developing normally. Her mother might want to get her evaluated, the teacher said. In March 2023, Mei was diagnosed with global developmental delay, a broad label with many causes. Specialists explained that some of Mei’s behaviors—the funny sounds she liked to make…
It says so much about how our society treats the neurodivergent that it's considered a non-extreme opinion that a dead child is preferable to an autistic one.
You should try being the parent of a seriously autistic child. I'd put a large chunk of cash down that most parents of severely autistic children wish their child had never been born. There's no upside, it basically ruins your life. Every interaction is a reminder of what you will never have, and what you will have to suffer through for the rest of your life.
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#55Earlier quoted context omitted.
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.
I think what OP was trying to ask was why would a parent be willing to put their child at lethal risk, using an experimental treatment, for a condition that itself is not lethal? I don't have a kid with autism or a learning disability so I don't know how I'd handle it, but if I did, I'd probably choose to live with it and help the child make the best of it. I would not be reaching for someone's research project.
Parents put their children at lethal risk (albeit small) for non-lethal conditions all the time.
Wisdom teeth removal under general anesthesia is around 1 in 100,000.
It doesn’t sound so crazy if the risks were presented as “only 10x as risky as wisdom teeth extraction”.
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#56An article about the complicated issue that is a child with a non-lethal developmental disorder getting a treatment that ends with the tragedy of the headline. The article might be sensationalizing the situation, but it makes the doctor out like a monster and as the facts read I can't say I disagree. That the treatment was inconclusive in the animal studies isn't particularly shocking. The brain is one of the more co…
I didn't read it as making the doctor a monster. What I saw in the story is an example of how the road to hell is paved with good intentions.
He's pretty clearly a monster or at least monster adjacent. He and his team
* Took hundreds of thousands of dollars under the table to develop the treatment
* Neglected to take steps that would determine whether the treatment was safe to use on humans
* Didn't inform the family that the treatment could possibly kill their daughter
* After the treatment did kill their daughter, he published the study anyway except 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.
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#57"Seven days after the girl’s medical team infused trillions of viruses carrying the recipe for the base editor into her spinal fluid, she died of a severe immune reaction linked to the therapy"
questions of a layman - couldn't they initially do a small infusion of the [may be even weakened version of that] viruses to check for the immune reaction? May be such infusion would really serve as a vaccine prepping the 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?
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#58I'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.
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
#59> When Mei was 4, one of her kindergarten teachers pulled Linda aside: Mei didn’t draw or write as well as the other kids and her language skills weren’t developing normally. Her mother might want to get her evaluated, the teacher said. In March 2023, Mei was diagnosed with global developmental delay, a broad label with many causes. Specialists explained that some of Mei’s behaviors—the funny sounds she liked to make…
It says so much about how our society treats the neurodivergent that it's considered a non-extreme opinion that a dead child is preferable to an autistic one.
The article clearly points out that it wasn't autism for which Mei was treated, but Snijders Blok–Campeau syndrome, of which autism is just a single symptom. That goes way beyond neurodivergency.
Re: Couple pay >$800k for a gene-editing therapy for their daughter. She died.
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