Gene therapy is at the initial stage of its development, so it makes sense to monitor patients whose DNA has been modified using the innovative CRISPR technique. However, for some scientists in China, this is obviously not a priority.
The Wall Street Journal reports that an unknown number of Chinese cancer patients who have undergone experimental gene therapy are not properly tracked, as one would expect. In these cases, patients have modified their genes using the CRISPR-Cas9 gene editing tool to treat cancer. According to the WSJ, scientists responsible for at least one study could not keep in touch with their patients afterwards and conduct subsequent studies.
Indeed, follow-up observations are extremely important for patients undergoing gene therapy. Changes in DNA can cause unintended effects, known as side effects. Unexpected health problems associated with gene modifications, such as autoimmune disorders, may appear later in life.
“Because we do not fully understand the human genome and are still developing [CRISPR-Cas9 and related technologies]we need to track perceived and unintended consequences throughout the lives of patients, ”said WSJ Jennifer Dudna, a biochemist at the University of California at Berkeley and co-author of CRISPR.
This is the latest alarming development for biomedical research in China. Last month, Chinese scientist He Jiankui announced that he had brought to life the world's first children with genetic editing. A scientist who works at the Southern University of Science and Technology in Shenzhen, said he used CRISPR to modify the DNA of human embryos, which led to the birth of twin girls with obvious immunity to HIV. Shortly after the news came, the Chinese government expanded its social lending system to include violations committed by researchers in order to curb local scientific misconduct.
Editing the human germline genes and embryo implantation into the mother’s womb is not yet allowed in China or anywhere else for that matter, mainly because gene editing is still at an incipient stage and because the altered traits will be inheritable (as in the US and in other countries). China, you can modify the embryos, but after a few days they need to be destroyed). On the other hand, editing of somatic genes, in which the DNA of a living person changes to treat various diseases, from cancer to hemophilia, leads to genetic changes that are not inherited. But somatic gene therapy, like a germline, is also in its infancy, requiring due diligence, responsible supervision, and tremendous caution.
Somatic gene therapy is legal in both China and the United States. In the US, research scientists are cautiously and reverently moving in this direction, and the Association for the Control of Food and Drug Administration is closely monitoring this. To date, only one gene therapy has been approved in the United States – a clinical trial at the University of Pennsylvania to test the safety of CRISPR with only 18 patients.
China has already edited 86 people with CRISPR
In the United States, the first scheduled clinical trials of human CRISPR gene editing begin. In the meantime, China is rapidly moving ahead, having already used the gene alteration tool to alter the DNA of dozens of people in several clinical trials.
In China, however, there is no equivalent to the FDA. The WSJ reports that doctors can proceed to clinical trials after receiving approval from their hospital ethics advice. As of January 2018, at least 86 patients in China have edited their DNA using CRISPR. The WSJ indicates that most of these tests are conducted by the privately owned company Anhui Kedgene Biotechnology Co.
According to someone familiar with this issue, one of Kejen’s projects has lost touch with patients whose DNA has been altered. Kedgene founder Mandy Zhou said that one study did not complete the study as planned, and as a result lost contact with patients. In this study, no patient died during treatment, she added.
According to Wang Yong, who conducted the study, another 18 patients were treated in Anhui Provincial Hospital in another study, Kejen. According to Dr. Wang, many participants died as they developed cancer, without specifying a specific number. Dr. Wang said the science ministry asked him to send a report on the trial this month when the Beijing authorities first asked for information about him, since it began more than a year ago.
Recently, three doctors who participated in gene editing tests were approached by representatives of the ministries of science and health of China (the closest thing in China is to the FDA, which in fact has not even been established). When the WSJ contacted these ministries for more information, they declined to comment.
All of this is very frustrating and regrettable, mainly because this unfortunate scientific behavior creates a bad reputation for CRISPR and the entire perspective of gene editing. CRISPR and other gene editing technologies are ready to eliminate many diseases and even to enter the era of selection and improvement of human traits (for example, twins edited by genes with immunity to the AIDS virus – this is indeed a very good idea in principle – it is simply premature).
Unfortunately, the situation with Chinese scientists may affect public opinion against these promising biotechnologies, which are already contradictory.[The Wall Street Journal]