6-year-old girl dies after parents paid $860K for experimental therapy that shouldnt have gone to trial

In an effort to treat their daughter’s genetic developmental disorder, parents in China paid respected researchers millions in Chinese yuan, a total of $860,000 USD, for a new treatment that had never been used in humans.But instead of celebrating advances in her writing and language skills, the family is mourning the loss of their only child.Her treatment went forward even when experts say success was unlikely, animal studies showed red flags for safety, and doctors downplayed risks — including of death, which wasn’t ever mentioned explicitly to the family, a new report in Science says.Zilong Qiu, who led the research for Mei’s treatment, appeared to be obsessed with publishing research in a prestigious journal, hoping hers would be “the first brain-directed gene-editing therapy.”Months after her death, he got what he wanted: a paper in Nature, only it doesn’t acknowledge her at all.The report from Science is making waves in the research world, raising questions about research oversight and capitalizing on desperate families to fund shaky science.“This shouldn’t have gone to trial,” Steven Gray of the University of Texas Southwestern Medical Center, who develops viruses for gene therapy, told Science.He and other experts are calling for a review of all images and data in the Nature paper published earlier this year by Qiu.The family asked to remain anonymous, and asked to refer to their daughter as “Mei.”Mei had a rare mutation on her CHD3 gene, which plays an important role in early development.

Kids like her with Snijders Blok-Campeau Syndrome often have intellectual disabilities and impaired speech and language.Mei’s teachers told her parents that she was struggling to keep up with other students in writing and language development, but she still had a mild case.Given that her disorder was not going to get worse over time and was not fatal, it raises questions about why such drastic action was recommended by the experts her parents con...