Gene therapies are very much at their preliminary stages of development, so it would make sense to keep tabs on patients whose DNA has been modified via the innovative CRISPR technique. For some scientists in China, however, this is apparently not a priority.
The Wall Street Journal is reporting that an undisclosed number of Chinese cancer patients who have undergone experimental gene therapies aren’t being properly tracked as would be expected. In these cases, the patients had their genes modified with the CRISPR-Cas9 gene-editing tool in an effort to treat their cancer. The scientists in charge of at least one trial failed to maintain ties with their patients afterwards and conduct follow-up examinations, according to the WSJ.
Indeed, follow-ups are extra critical for patients undergoing gene therapies. Changes to DNA can trigger unintended consequences known as knock-off effects. Unexpected health problems stemming from gene modifications, such as autoimmune disorders, could appear later in life.
“Since we do not fully understand the human genome and are still developing knowledge of [CRISPR-Cas9 and related technologies], we need to monitor the intended and unintended consequences over the lifespan of patients,” Jennifer Doudna, a biochemist at the University of California, Berkeley, and a co-inventor of CRISPR, told the WSJ.
This is the latest troubling development for biomedical research in China. Last month, Chinese scientist He Jiankui claimed to have produced the world’s first gene-edited babies. The scientist, who works at Southern University of Science and Technology in Shenzhen, said he used CRISPR to modify the DNA of human embryos, resulting in the birth of twin girls with an apparent immunity to HIV. Shortly after that news broke, the Chinese government expanded its social credit system to include infractions made by researchers, an effort to curb endemic scientific misconduct.
Human germline gene editing and the implantation of embryos into a mother’s womb is not yet legal in China or anywhere else for that matter, mostly because gene-editing is still in its nascent stages and because modified traits would be heritable (in both the U.S. and China, it’s okay to modify embryos, but they have to be destroyed after a few days). Somatic gene editing, on the other hand, in which a living person’s DNA is altered to treat various maladies, from cancer through to hemophilia, results in genetic changes that are not heritable. But somatic gene therapies, like the germline variety, are also in their infancy, requiring due diligence, responsible oversight, and a hefty amount of caution.
Somatic gene therapies are legal in both China and the United States. In the U.S., research scientists have tread carefully and trepidatiously in this direction, with the Food and Drug Association maintaining a watchful eye. To date, only one gene therapy has been approved in the U.S.—a clinical trial at the University of Pennsylvania to test the safety of CRISPR and involving just 18 patients.
In China, however, there’s no equivalent to the FDA. Doctors can proceed with a clinical trial after receiving approval from their hospital’s ethics boards, the WSJ reports. As of January 2018, at least 86 patients in China have had their DNA edited with CRISPR. Most of these trials are being conducted by Anhui Kedgene Biotechnology Co., a private startup, as the WSJ points out:
One of Kedgene’s projects has lost touch with patients whose DNA was altered, according to a person familiar with the matter. Kedgene founder Mandy Zhou said one trial didn’t complete the research as planned, and as a result lost touch with patients. No patients died during treatment in that trial, she added.
Another Kedgene trial, at the Anhui Provincial Hospital, treated 18 patients, according to Wang Yong, who ran it. Many participants died as their cancer grew, Dr. Wang said, without giving a specific number. Dr. Wang said he was asked by the science ministry this month to send a report on the trial, the first time authorities in Beijing sought information about it since it began more than a year ago.
Three of the doctors involved in the gene-editing trials were recently contacted by China’s science and health ministries (the closest thing that China has to the FDA, which isn’t really even close). When the WSJ contact these ministries for more information, they declined to comment.
This is all very frustrating and regrettable, mostly because this deplorable scientific behavior is giving CRISPR and the entire prospect of gene-editing a bad name. CRISPR and other gene-editing technologies are poised to eliminate scores of diseases, and even usher in the age of human trait selection and enhancements (the gene-edited twins with an immunity to the AIDS virus, for example, is actually a very good idea in principle—it’s just grossly premature).
Sadly, the situation with China’s scientists could sway public opinion against these promising biotechnologies, which are already controversial.