Gene therapies are at a very early stage of development. It would therefore be useful to observe patients whose DNA has been modified by the innovative CRISPR technique. However, for some scientists in China, this is obviously not a priority.
The Wall Street Journal reports that an undisclosed number of Chinese cancer patients who have undergone experimental gene therapy are not expected to perform as expected. In these cases, patients have modified their genes with the CRISPR-Cas9 gene editing tool to treat their cancer. The scientists responsible for at least one study were unable to maintain contact with their patients and conduct follow-up, according to the WSJ.
In fact, follow-ups are particularly critical for patients undergoing gene therapy. Changes to the DNA can trigger unintended consequences called repulsion effects. Unexpected health problems due to gene alterations such as autoimmune diseases could occur later.
"Because we do not fully understand the human genome and are still developing knowledge about [CRISPR-Cas9 and related technologies]we need to monitor the intended and unintended consequences for patient lifetime," said Jennifer Doudna, biochemist at the University of California, Berkeley, and co-inventor of CRISPR the WSJ.
This is the recent worrying development for biomedical research in China. Last month, Chinese scientist He Jiankui claimed to have produced the world's first born babies in the world. The scientist, working at the Southern University of Science and Technology in Shenzhen, said he used CRISPR to modify the DNA of human embryos, leading to the birth of twin girls with apparent immunity to HIV. Shortly after the news broke, the Chinese government expanded its social credit system to include violations of researchers to stem endemic scientific misconduct.
Humane germ line gene editing and implantation of embryos into the womb is not yet legal in China or anywhere else, especially as gene editing is still emerging and modified characteristics are inheritable (in both cases) In the USA and China, it is okay to modify embryos, but they will have to be destroyed after a few days. On the other hand, somatic gene editing, in which the DNA of a living person is altered to treat various diseases, from cancer to hemophilia, leads to genetic changes that are not inheritable. But somatic gene therapies, like the germ line variety, are still in their infancy and require due diligence, responsible supervision and a high degree of caution.
Somatic gene therapies are legal in both China and the US. In the US, researchers have carefully and boldly gone in that direction, with the Food and Drug Association keeping a close eye on it. So far, only one gene therapy has been approved in the US ̵
China already has 86 people with CRISPR 
In the US, the first planned clinical trials for CRISPR gene processing in humans are about to begin …
In China, however, has no equivalent to the FDA. Doctors can proceed with a clinical study after the approval of the responsible ethics committee of their hospital. In January 2018, DNA was processed with CRISPR in at least 86 patients in China. Most of these studies are conducted by Anhui Kedgene Biotechnology Co., a private startup company, as the WSJ points out:
One of Kedgene's projects has lost contact with patients whose DNA has been altered, according to one person, who is familiar with the matter. Kedgene founder Mandy Zhou said a study did not complete the research as planned, thereby losing contact with patients. No patient died during treatment in this study, she added.
Another Kedgene study at Anhui Provincial Hospital treated 18 patients, Wang Yong, who led them, said. Many participants died when their cancer grew, Dr. Wang, without naming a specific number. Wang said he would be asked this month by the Science Ministry to send a report on the trial when the authorities in Beijing first sought information since they began more than a year ago.
Three of the WHO Involved in the WAS Recently, studies on gene-editing tests have been conducted by China's science and health ministries (what China comes closest to FDA, which is not even that close). When the WSJ contacted these ministries for further information, they refused to comment.
This is all very frustrating and unfortunate, especially because this unfortunate scientific behavior gives CRISPR and the entire gene editing perspective a bad name. CRISPR and other gene editing technologies are able to eliminate a variety of diseases and even initiate the age of selection and enhancement of human characteristics (eg, gene-edited twins with immunity to the AIDS virus are one very good idea) in principle – it is only roughly premature.
Unfortunately, the situation with China's scientists could influence public opinion against these promising biotechnologies, which are already controversial.
[The Wall Street Journal]