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

ncallaway 3 hours ago | parent | next [-]

> 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.

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cogman10 3 hours ago | parent | next [-]

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.

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neonstatic 3 hours ago | parent [-]

The part about gifts he received from the father in person is particularly vile.

	▲	rainprincess 2 hours ago \| parent [-]
		I think it's a part of the culture of guanxi (relations) in China.

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dmix 3 hours ago | parent | prev [-]

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...

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ncallaway 3 hours ago | parent [-]

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?

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dmix 3 hours ago | parent [-]

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 2 hours ago \| parent [-]
		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

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fabian2k 3 hours ago | parent | prev | next [-]

It's worse than that. The therapy likely killed her, and her condition wasn't fatal in the first place.

	▲	asqueella 3 hours ago \| parent \| next [-]
		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.
	▲	processing 3 hours ago \| parent \| prev [-]
		"global developmental delay" yeah this is tragic - this kid didn't need to go through this.

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3 hours ago | parent | prev | next [-]

[deleted]

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slashdave 2 hours ago | parent | prev [-]

https://en.wikipedia.org/wiki/Hippocratic_Oath