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FFor years, we have speculated on the promise that CRISPR – a gene editing tool that can accurately modify DNA sequences – can treat human diseases. This week, our high expectations have been put to the test. On Wednesday, reports confirmed that the US has embarked on a new clinical trial using CRISPR to treat cancer patients, the first of many to come.
2019 is the year when the drive wheels come off.
CRISPR is a powerful and controversial tool, partly because it is not clear its safety and reliability, in part because it can be used, create genetic changes passed down from generation to generation, if used in germ cells such as eggs and sperm. In general, it functions as a cut-and-paste function of a word processor, cutting specific DNA fragments that researchers can replace with new sequences. In this way, scientists can suppress carcinogenic mutations of DNA and replace them with DNA sequences that boost the immune system, as do scientists at the University of Pennsylvania in their new essay .
As NPR According to the information provided, the team used CRISPR for the genetic engineering of the immune cells of two cancer patients, one with multiple myeloma and the other with sarcoma. According to the clinical trial literature, their goal is to create samples of T cells (immune system cells) from each patient using CRISPR to suppress one gene and add another gene, thus providing the immune system with new tools to combat the disease. cancer in their body.
This lawsuit is a decisive moment for the United States. However, this is not the first time that CRISPR is used in humans. To the horror of the international genetics community, in late 2018, Chinese scientist He Jiankui used CRISPR to edit human embryos, in hopes of making twin twins resistant to HIV. Many clinical trials using CRISPR on cancer cells have taken place in China, and a few have started in Europe and Canada.
The new clinical trial is very different from the experiments performed so far. The purpose of this trial was to conduct the CRISPR test in cancer patients in an ethical and conservative manner, and it is already paving the way for many other similar trials in the United States.
How was this clinical trial approved?
The project currently underway at the University of Pennsylvania was first proposed in 2016. It took nearly two years for the work to begin, with federal agencies weighing the risks and benefits. In contrast, China's CRISPR trials at the Hangzhou Cancer Hospital were approved after a single afternoon.
… The authorities overlap and are probably redundant. "
American scientists have proceeded with care with regard to CRISPR and other gene editing technologies because of security issues and because of the ethical implications of editing human genetic material. The process was slow because historically, clinical trials of gene therapy had to be approved by the Food and Drug Administration. and followed by the National Institutes of Health's Recom- mended DNA Advisory Committee (RAC), which would review all protocols. Together, these agencies have formed a dual layer of control because gene editing has long been considered an unknown territory.
However, in 2014, things started to change.
In an announcement, NIH Director Francis Collins said that she would allow the RAC to override the FDA in regulating gene therapy trials. "With the evolution of science, the oversight roles of the RAC, US Food and Drug Administration and institutional oversight authorities overlap and are undoubtedly redundant," Collins said in 2014.
The CRISPR clinical trial process has been further simplified since the efforts of Collins and former FDA Commissioner Dr. Scott Gottlieb. Write in the New England Journal of Medicine in August 2018, they described CRISPR as a "qualitative leap" for the sector, stressing that the regulations would change to "accelerate progress safely":
In the opinion of senior officials of the FDA and NIH, there is more evidence to say that the risks of gene therapy are quite unique and unpredictable – or that the field still requires special monitoring that goes beyond the existing framework to guarantee security.
Just two days after the publication of their statement to the newspaper NEJM, they submitted to the Federal Register a notice deleting the CAR revision and deleting the NIH reporting requirements for human gene therapy testing.
In doing so, two major regulators have made it clear that gene therapy clinical trials are no longer as radical as we thought.
More trials in progress
On September 5, 2018, Penn's clinical trial recruited his first patient, according to official documents. Importantly, it is unclear whether the new policy changes have anything to do with the approval of the trial. Nevertheless, as these processes unfolded simultaneously, they indicated that the US was ready to truly delve into CRISPR, both scientifically and in regulatory terms.
The NIH Global Clinical Trial Database lists 26 trials worldwide involving the use of CRISPR in human cells. They are all at different stages: some recruit, others are suspended. Twelve of these trials are in China and nine are in the United States.
This suggests that gene therapy is becoming a more normal aspect of the search for a new treatment by the FDA – or at least that it does not require additional agencies to monitor it carefully all the time. Talk to NPRFyodor Urnov, deputy director of the Altius Institute for Biomedical Sciences in Seattle, also said it was already a great year for the use of CRISPR in humans.
"2019 is the year when the training wheels stand out and the world sees what CRISPR can really do for the world in the most positive sense," he said.
Trying to be cautious about the alarming uses of this powerful technology, the US appears to be modernizing its regulatory frameworks to allow scientists to probe the good that CRISPR can do. This clinical trial in Penn, which should be completed by 2033, is one of the first to do so – but it will certainly not be the last.
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