An experimental gene therapy killed a 6-year-old girl in China. Her parents are seeking accountability.

An investigation by Science and Retraction Watch has uncovered the tragic death of a 6-year-old girl in China following an experimental gene therapy procedure. The child suffered from Snijders Blok-Campeau syndrome, a condition that is not typically fatal, yet the treatment resulted in her death just seven days after the injection.

The therapy was conducted at Xinhua Hospital, part of Shanghai Jiao Tong University School of Medicine. Records show that the family contributed the equivalent of $860,000 for the development and deployment of the treatment. Despite pre-clinical animal studies showing signs of severe liver and kidney damage in monkeys, the procedure moved forward without additional safety reviews.

Following the girl's death, the hospital received a minor fine for failing to register the research as a clinical trial. The neuroscientist leading the project, Zilong Qiu, later published a paper on the work in the journal Nature, which initially excluded mention of the patient's death or the family’s financial contributions. The parents have since campaigned for the retraction of that study, raising serious questions about the oversight and regulation of experimental gene therapies in China.

This case highlights the risks associated with unregulated medical experiments and the lack of transparency in reporting patient outcomes. Experts note that the absence of strict ethics board oversight, combined with private financial arrangements, created a dangerous environment for this young patient.