China takes lead in CRISPR gene-editing race with first human trials

November 18, 2016

Taking the lead in using the groundbreaking gene-editing technology CRISPR-Cas9 in human trials, scientists at Sichuan University in Chengdu, China are the first in the world to inject CRISPR-modified cells into a patient with aggressive lung cancer in an effort to make the patient’s immune system more effective at combating cancer cells.

The researchers, led by oncologist Lu You, said that the first round of treatment in a safety trial went well enough to warrant a second injection of the Cas9-edited cells for the patient. Nine more people are already slated to undergo the treatments in the future, who will each receive either two, three or four injections.

From there, the patients will be monitored for at least six months, if not longer, to “determine whether the injections are causing serious adverse effects.”

CRISPR is a recently emerged technology that acts like a tiny pair of molecular scissors that can cut and alter nucleotides which make up the DNA, enabling scientists to find and modify or replace genetic defects.

The team removed immune cells from the recipient’s blood and then disabled a gene in them using CRISPR-Cas9. The disabled gene codes are for the protein PD-1, which normally stops a cell’s immune response. Cancer cells take advantage of PD-1’s function to proliferate.

Lu’s team then cultured the edited cells, increasing their number, and injected them back into the patient, who has metastatic non-small-cell lung cancer. The hope is that, without PD-1, the edited cells will attack and defeat the cancer.

The Chinese trial is expected to trigger a global race to carry out human trials of the groundbreaking medical technology throughout the world.This US is already expected to conduct its own cancer fighting trials next year.

Carl June, from the University of Pennsylvania in Philadelphia, thinks that the Chinese trial will trigger something like a “Sputnik 2.0”,  biomedical duel between China and the US.

June is the scientific adviser for a planned US trial that will use CRISPR to target three genes in participants’ cells, with the goal of treating various cancers. He expects the trial to start in early 2017.

Meanwhile, a group at Peking University in Beijing also hopes to start three clinical trials using CRISPR against bladder, prostate and renal-cell cancers in March 2017.

The planned CRISPR trials do not have approval or funding yet.

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