Wu: What's the point of China taking the lead in starting genetic editing human trials?

In 2012, scientists from the United States and Austria worked together to improve the CRISPR-Cas9 system and foreshadowed in their published research paper that CRISPR could be used as an efficient and specific RNA-mediated gene editing tool.
2013, Zhang Feng et al. used CRISPR for gene editing of mammalian cells, ushering in a new era of CRISPR as a programmable gene editing tool.
, a leader in CRISPR technology, has yet to approve its use in human trials, but China has fewer regulatory restrictions, making it the first country to use CRISPR in human trials.
July 2016, The team of Professor of Oncology at Huaxi Hospital, Sichuan University, announced that it will conduct the "first case of crispR-Cas9 gene editing human trials" in the world.
March 2017, Wu Qixuan, director of Hangzhou Cancer Hospital, also began experimenting with gene editing techniques to treat cancer patients. On January 21,
, the Wall Street Journal published a lengthy article commenting on China's first effort to begin genetic editing of human trials, in which Wu reported on the experiment.
it is understood that Wu's team extracted blood samples from esophageal cancer patients, transported them to Anhui Corden Biotech Co., Ltd. laboratory through high-speed rail, using CRISPR technology to remove the genes in the blood's immune cells that affect tumor killing, and then put them back into the patient's body, hoping that modified immune cells can kill tumor cells, so as to achieve therapeutic purposes.
the trial was approved by the Ethics Committee of Hangzhou Cancer Hospital, whose members are appointed by the hospital and include several doctors, a lawyer and a patient. The
Committee approved the trial in just one afternoon, and because China's Health and Planning Commission has authorized the hospital ethics committee to review the clinical trial and assess the risk, Wu does not need to pass the relevant departments of the Commission.
, by contrast, the U.S. plans to conduct genetic editing human trials isn't really too late, but the vetting process is more rigorous.
researchers must first obtain approval from the U.S. Food and Drug Administration (FDA) before conducting human trials.
, the lead scientist for the first gene-edited human trial in the United States and the lead scientist for the CRISPR research team at the University of Pennsylvania, first attempted to get an assessment from the National Institutes of Health (NIH) advisory board in 2016. The
committee has made a series of demands, such as describing the experiment as "gene transfer" rather than "gene therapy" to ensure that patients know it is an experiment rather than a treatment, and to conduct various tests to observe CRISPR's off-target.
, which was eventually approved by the NIH, June's team spent another year discussing with the FDA, providing it with information and answering questions.
it is understood that they are currently awaiting final FDA approval, which is expected to be approved as early as this month.
but this is not the end, and even if the trial begins, Penn will still face more stringent standards than Wu.
, Penn researchers had to use an informed consent form approved by the FDA and the hospital evaluation committee, which only briefly mentioned genetic engineering, nor did they detail the experimental tools they were using.
, according to a related staff at Cotton Biotech, Chinese patients will also sign consent signed, but are basically listening to doctors.
for serious adverse events such as deaths during the trial, an FDA spokesman said, regardless of whether the death is related to the trial, the test conductor must report to the FDA, and the Chinese Health and Planning Commission requires researchers to report adverse events to the hospital ethics committee.
, Wu's trial has so far had a case of death, and he said the death was caused by the disease, which has nothing to do with the trial itself, so it does not need to be reported.
In addition, one of the main purposes of the new drug Phase I clinical trial is to initially assess the human safety of the drug.
June said Penn will first test CRISPR on one patient and wait a month to make sure no adverse reactions occur before testing on two other patients.
Wu believes that saving the lives of patients is the most important, he has now edited more than 10 cancer patients' genes, in the future plans to test more patients.
, the U.S. National Library of Medicine database has recorded nine trials in China, and the actual number may be more than that.
China is striving to bring domestic industries on the international stage, and gene editing is included in the national five-year plan drawn up in 2016.
June also said that China could overtake the United States in applying medical technologies pioneered by Western countries such as CRISPR, which could lose its leading position in biomedicine in the future;" The battle between China and the U.S. over CRISPR could trigger a 1950s-era U.S.-Soviet space technology competition, which will be version 2.0.
" commented that while policies on human trials may differ, China's bold attempts have raised concerns among Western scientists about the safety of the new tool, and they do not believe the U.S. should relax its regulations. In an interview
, Laurie Zoloth, a bioethicist and dean of the University of Chicago's Theological Seminary, said she wants countries to develop uniform standards and share test results and ethical guidelines.
in the immature field of gene editing, it is still difficult to find the ideal balance between rapid propulsion and safety assurance.
, Stanford University's Matthew H. Porteus team found that in most people, there is already a body fluid immunity and cell immunity against the Cas9 protein, crispR-Cas9 technology in the human body can trigger a serious immune response.
Wu agrees that CRISPR technology is a double-edged sword, but says that when others pay more attention to potential risks, they see potential efficacy.
terminal cancer patients on the front line of life and death could not allow him to spend too much time on tests, "you never know the results without trying."
" in the PLA 105 Hospital and the Hangzhou Cancer Hospital trial, the relevant personnel said that some patients' condition has been alleviated, but so far, China's various gene editing human trials have not announced the results.
china and the United States have invested heavily, six companies are listed around the world, the next billion market is in the middle of january 2018, the United States announced that it will invest $190 million over the next six years to support somatic gene editing research to develop safe and effective gene editing tools to treat more human diseases.
the United States at great expense to develop gene editing technology, obviously very optimistic about this technology.
in the past two years, China's policy in the field of precision medicine has been very intensive, giving a lot of support to technologies such as gene editing.
, for example, to raise precision medicine as a national strategy, with plans to invest 60 billion yuan by 2030.
issued the "13th Five-Year Plan" for Biotechnology Innovation in April 2017 to identify the development of "next-generation gene manipulation technology".
specific ally to develop accurate or quantitative new gene operation technology, eukaryotes cell gene (group) editing technology, in industrial production and environmental protection and other aspects of important application value of new microbial gene recombination technology, promote the integration of a variety of gene (group) editing means, pay attention to the efficiency and flux of gene operation, improve ease of operation, reduce off-targeting, expand the scope of application.
According to a new report by the China Business Industry Research Institute, the global precision medicine market will grow at a rate of 15% per year from 2016 to 2020.
the global precision medicine market is expected to reach $105 billion by 2020, and "gene scissors" will be the key to prying the market of hundreds of billions.
the global development of the shell society combed the foreign gene editing related enterprises (below table), from the table can be seen that 17 companies have been listed, and most of the headquarters in the United States, the United States is still in the forefront of gene editing technology.
the technology used in each country also covers almost three generations of editing technology, but still crispR-based, in addition to adenovirusor or adeno-related virus technology is more common, followed by the emergence of CAR-T joint CRISPR technology, indicating that the combination therapy will become a new trend.
finally, Associate Professor Han Chunyu of Hebei University of Science and Technology claims to have discovered a new nucleic acid enzyme, Argonaute, which is different from CRISPR, which has attracted global attention and has attracted Denmark's Novozymes to seek cooperation, but the findings have not been recognized or even suspected to be academic fraud.
China become a strong rival to the United States Although the United States gene editing level in the world's leading, but China's technology in this field is also the world's leading, the U.S. media even said that "China's gene editing technology development makes the United States out of reach."
January 23, 2018, media reported that The director of Hangzhou Cancer Hospital and oncologist Wu Qixuan has been trying to treat esophageal cancer patients with CRISPR-Cas9 technology since March last year.
China, the first country in the world to use CRISPR for human trials, has so far received gene-editing treatment in at least 86 Chinese patients.
the main reason why the U.S. lost its lead is that the U.S. has more stringent regulation, needs to pass a variety of risk assessments and safety checks, in the best possible care of patients under the premise of the implementation of gene editing treatment.
there is no doubt that China will be a strong rival to U.S. gene-editing technology.
incomplete statistics, China involved in gene editing enterprises have 21, most of them to technical support and service outsourcing mainly, customer-sourced medical institutions, enterprises, research institutes and universities, etc. , it can be seen that gene editing technology itself in the domestic development is more mature, higher acceptance.
.