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.

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

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

> 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.

> 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.

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.

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.

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.

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.

"The young girl tugged on her mother’s hand as they pressed through the doors ..."

Zack-D films tier writing there, disgusting.

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

"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"

as a layman - couldn't they initially do a small infusion of the 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?

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.