The girl, who bore the pseudonym “May”, died a week after receiving a spinal injection containing trillions of viruses.
WASHINGTON: A six-year-old Chinese girl died last year after receiving experimental gene therapy to correct a non-life-threatening condition, according to an investigation by Science and Retraction Watch.The report said Thursday that the girl, who bore the pseudonym “May,” died a week after receiving a spinal injection of trillions of viruses last March, designed to penetrate the brain and repair a rare genetic condition that caused her mild cognitive impairment. But the report said her parents, who paid more than $800,000 to fund the experiment, were not adequately informed of the risks.The lead researcher went on to publish a paper on the relevant animal studies in Nature, which did not mention May, noting that “bridging the gap between preclinical research and clinical translation remains a major challenge.”May had a rare genetic condition called Snyder-Block-Cambo syndrome, which is known to affect only 237 people worldwide. The disease is caused by a single mutated DNA base, a T that should be a C, and most affected individuals have a normal life expectancy, have larger-than-normal heads and intellectual deficits, although the extent varies greatly.The trial was led by neuroscientist Zilong Qiu, one of many researchers globally racing to turn base editors — a more precise form of CRISPR gene editing — into personalized treatments for children with rare genetic conditions.
Seven experts who reviewed the details said Chiu and his team played down the risks and moved forward despite the low odds of success. They also called for a full review of the data submitted to Nature, saying some findings may warrant retraction. This case also raises questions about whether these treatments should initially be tested in patients with non-fatal conditions.(This is an AFP story)
