Paper Concealed Death of 6-Year-Old Daughter...Controversy over Gene-Editing Clinical Trial in China

Joint Investigation by Science and Retraction Watch

Clinical Trial Proceeded Despite Knowledge of "Liver Damage in Four Monkeys"

Special Investigation Launched by Shanghai Jiao Tong University

A prominent Chinese scholar has come under fire after it was revealed that he published a paper in an international academic journal without disclosing the death of a six-year-old patient during a brain gene-editing clinical trial.

The international academic journal Science and "Retraction Watch" disclosed the death of a young girl that occurred during a clinical trial conducted at Xinhua Hospital affiliated with Shanghai Jiao Tong University, China. Official website of Science

According to AFP and China's state-run Global Times on the 27th (local time), Shanghai Jiao Tong University School of Medicine announced the previous day that it has formed a special investigation team to look into the clinical trial conducted at its affiliated Xinhua Hospital. The university stated, "We firmly oppose any medical research that violates research ethics and regulations," adding that it will take necessary measures in accordance with the results of the investigation.

The death was first made public on the 23rd through a joint investigative report by the science journal Science and the research ethics watchdog group Retraction Watch. According to their findings, a six-year-old girl referred to as "Mei" (a pseudonym) was suffering from Snijders Blok-Campeau syndrome, caused by a CHD3 gene mutation. This disorder is accompanied by developmental delays, speech impairments, and autism symptoms, but it is not considered immediately life-threatening.

Parents Provided 1.2 Billion Won in Funding: "We Didn't Know How Dangerous It Was"

Image to assist understanding of the article. Pixabay

Mei's parents, in search of a treatment for their daughter, were introduced to Xinhua Hospital through a patient support group. Zhu Zilong, a neuroscientist at Shanghai Jiao Tong University, persuaded the parents by explaining that he was developing the world's first gene-editing therapy targeting the brain. The parents subsequently raised funds by borrowing from relatives, ultimately providing a development budget of $860,000 (about 1.26 billion won).

In March last year, Professor Zhu's research team injected a viral vector containing a base-editing agent into Mei's spinal canal. The method was designed to reach brain nerve cells through the cerebrospinal fluid flowing in the spinal cord. However, Mei experienced a severe immune reaction and died within a week.

The problem was that there were already warning signs of risk. In prior toxicity tests conducted on monkeys, all four subjects exhibited moderate to severe liver damage, and one monkey treated with a high dose also showed kidney damage. However, the Xinhua Hospital Ethics Committee approved the clinical trial in January last year without reviewing the final toxicity test report.

Mei's parents told Science, "At the time, we did not realize how unusual and dangerous some of the procedures were." Experts in gene therapy and bioethics who reviewed the case have raised concerns, questioning whether the risks were properly communicated to the parents.

Paper Concealed Death...Hope Amid Report Leads to Disclosure of the Truth

In February, the research team published the results of the preclinical study of this therapy in the international journal Nature. The paper highlighted the successful mouse experiments funded by Mei's family, but omitted any mention of Mei's death. The paper was praised by some scientists, and China Central Television (CCTV) even reported it as "a ray of hope for families suffering from rare diseases." It is reported that this broadcast prompted Mei's family to decide to go public with the incident.

The Chinese regional health authorities fined Xinhua Hospital $3,600 (about 5.28 million won) last September for failing to properly supervise the clinical trial and for failing to register it in the national database as an industry-sponsored study. However, Professor Zhu was not subject to any penalties. Professor Zhu did not respond to inquiries from Science, and told the South China Morning Post (SCMP) that "all answers will be provided by the medical school leadership."

Science noted that after the 1999 death of 18-year-old Jesse Gelsinger in an early gene therapy clinical trial in the United States, research in the field was scaled back for nearly a decade. The journal questioned whether dangerous treatments should first be tried on patients with non-lethal diseases. It also pointed out that while studies funded by Chinese government agencies are subject to stricter oversight, the process in this case was lax because it was funded privately.

This content was produced with the assistance of AI translation services.