(Bloomberg) — Chinese scientists are embarking on what appear to be the first human trials with the Crispr gene editing tool, the latest effort by the country’s researchers to master a technology that might someday be a potent tool in developing therapies worldwide.
The group led by Lu You, an oncologist at the south-western Sichuan University West China Hospital, has recruited the first patient for a ten-people clinical trial, which will activate immune cells using Crispr and infuse them back into patients to fight lung cancer. Due to potential risks in using the pioneering treatment for humans, the team has decided to treat the first group of three patients one at a time, Lu said in a phone interview.
Formally called Crispr-Cas9, the genetic editing tool acts like a pair of precise molecular scissors that can cut out unwanted sections of DNA and insert desired ones. The team is using it to remove a gene that encodes a protein named PD-1, which normally keeps the immune cells in check but is also used by cancer cells to hide from the immune system.
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The engineering is intended to switch on the immune response to attack cancer. In the pharmaceutical industry, antibody drugs directly blocking the PD-1 protein including Merck & Co.’s Keytruda and Opdivo sold by Bristol-Myers Squibb Co. have become new growth engines for the companies.
“If this technology has good safety and shows certain efficacy, it has wide applications,” said Lu. If successful, it has the potential to become an alternative biotechnology treatment to replace or complement the existing single antibody drugs, Lu said. Lu’s group received approval from the ethics board of the West China Hospital, one of the top facilities in the country.
They will observe the first patient for two months after injecting the Crispr-edited cells and if there is no problem, proceed to give the second patient the same treatment. If the first group of three patients responds well, they may be able to accelerate after that.