Chinese scientists will be the first to use the CRISPR, a gene editing tool, on humans. A team of Chinese researchers will be led by Lu You, an oncologist at the south-western Sichuan University West China Hospital.

The clinical trial will involve 10 patients with lung cancer. In the trial, immune cells will be activated using CRISPR and then would be put back into patients to treat lung cancer. There is risk associated with the treatment, which is never tested before on humans. At first, it will be performed on a group of three patients, one at a time. The ethics board of the West China Hospital, one of the top facilities in the country, gave approval to the trial.

CRISPR-Cas9 is a genetic editing tool that edits DNA to make them able to fight certain defect in the body. It basically removes something from the gene and adds something else to get the desired results. In case of the upcoming trial, the team would use the tool 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.

It is processed to turn on the immune response to attack cancer. In the pharmaceutical industry, there are antibody drugs that directly block the PD-1 protein including Merck & Co.’s Keytruda and Opdivo that are sold by Bristol-Myers Squibb Co. Such drugs are helping companies make great profits.

“If this technology has good safety and shows certain efficacy, it has wide applications”, said Mr. Lu. If the trial is successful, the treatment devised could replace or compliment antibody drugs as an alternative biotechnology treatment.

A report published in Post-Gazette revealed, "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."

“If this technology has good safety and shows certain efficacy, it has wide applications,” Mr. Lu said. If successful, it has the potential to become an alternative biotechnology treatment to replace or complement the existing single antibody drugs, Mr. Lu said. His group received approval from the ethics board of the West China Hospital, one of the top facilities in the country.

“We’ve learnt from past lessons that safety comes first. You have to make clear its safety profile before its preliminary efficacy can be discussed,” Mr. Lu said. Patients must have the PD-L1 bio-marker and should have tried three to four lines of existing treatments available in China and failed to benefit from them, he said.

According to a report in Fierce Biotech by Ben Adams, "In an official announcement made yesterday, Chinese researchers, led by You Lu of Sichuan University, plans from next month to begin the world’s first CRISPR trial at the West China Hospital, Sichuan University, after being cleared by the hospital’s ethics board."

The lymphocytes will be selected and expanded ex vivo and then infused back into patients. Cyclophosphamide at 20 mg/kg single dose will be administered 3 days IV before cell infusion. Interleukin-2 (IL-2) will also be given in the following 5 days, with patients getting a total of two, three or four cycles of treatment.

But exactly one month ago today, in a slightly bizarre twist, the co-founder of Napster and first president of Facebook, Sean Parker, threw his weight and wallet behind T-cell biology specialist Dr. Carl June, and a series of major U.S. medical academic centers led by the University of Pennsylvania, in an effort to get their team to run the first-ever CRISPR trial.