Researchers at Sichuan University's
West China Hospital has announced plans to begin a clinical trial where cells
modified using the CRISPR gene editing technique will be used on human beings
for the very first time. They plan to edit genes in such a way as to turn off a
gene that encodes for a protein that has been shown by prior research to slow
an immune response and by so doing treat patients with lung cancer.
The CRISPR gene editing technique has been in the news a lot
of late as scientists creep ever closer to using it as a means to treat
diseases or to change the very nature of biological beings. China has been a
leader in promoting such research on human beings—they were the first to use
the technique to on human embryos.
This new effort is seen as far less
controversial—a team in the U.S. is planning a similar study as soon as they
can get regulators to greenlight their project. The Chinese team plans to
retrieve T cells from patients that have incurable lung
cancer and then edit the genes in those cells. More specifically,
they will be looking to disable a gene that encodes for a protein called
PD-1—prior research has shown that it acts as a brake on an immune
response to help prevent attacks on healthy cells. Once the cells
have been edited and inspected very carefully to make sure there were no
editing errors they will be allowed to multiply and then all of the cells will
be injected back into the same patient's bloodstream. It is hoped that the
edited cells will cause the immune system to mount a more aggressive attack on tumor
cells, killing them and curing the patient.
The researchers acknowledge that they
do not know for sure how the body will respond, whether it will cause a more
aggressive attack on the tumor cells or kick off other problems related to an
overzealous immune response.
The clinical trial is set to start next
month, 30 candidates have been chosen, but only one will get the edited cells
initially—a three dose regimen. That patient will be monitored very closely for
both positive and negative responses—the overall goal is to see if the
procedure is safe, but the researchers are hoping, of course, to see some sign
of tumor reduction.
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