On October 28, 2016, China entered the history books as the first country ever to test the revolutionary yet controversial CRISPR gene editing technology on humans. Specifically, a research team from the Sichuan University led by oncologist Dr. Lu You inserted modified immune cells into a lung cancer patient at the West China Hospital.
The immune cells injected were the recipients’ own cells. These cells were extracted from the patient’s own blood, after which CRISPR-Cas9 was used to disable the gene that expresses the protein PD-1. CRISPR-Cas9 is a gene editing tool that can be programmed and instructed to target specific genetic codes, then modify or cut DNA at precise locations. PD-1 is the protein that normally stops a cell’s immune response, which is why when it’s active, cancer cells can proliferate uncontrollably.
After editing the cells, these were then cultured so they could multiply. Upon reaching a sufficient number, the cells were injected back into the patient. Without the PD-1 protein, the hope is that the modified cells will be able to attack and defeat the cancer.
In total, that particular clinical trial will be performed on 10 patients, with each patient receiving two, three or four injections of re-engineered cells. The focus of the study will be to determine the safety of the treatments, and whether there will be harmful side effects or not. Results are expected to be released this year.
Six months later, China is once again making history as researchers at the Nanjing University have just launched the second human clinical trials for CRISPR-Cas9. This time, the trial will be at Nanjing University’s Nanjing Drum Tower Hospital, and will be participated in by 20 patients with different types of cancer including aggressive gastric cancer, lymphoma and nasopharyngeal carcinoma — a rare kind of cancer affecting the head and neck.
Similar with the first trial, the aim is to use CRISPR to restrain specific genes from activating and preventing the patient’s immune system from working to fight the cancer.
As reported by The Wall Street Journal, Jia Wei — vice-director of Nanjing’s Clinical Cancer Institute and one of the leaders of the trial – said that the first patient (who is suffering from late-stage nasopharyngeal carcinoma) had already received the cell infusion on April 28, 2017 and so far, things seem to be going well.
On the other side of the world, the green-lighted first human CRISPR trial in the U.S. is expected to begin soon at the University of Pennsylvania. Designed to test CRISPR’s safety rather its effectivity in treating cancer, the test will involve 18 patients with various types of cancer. It will make use of the patients’ T cells (a type of immune cell), edited three times by CRISPR, prior to being injected back into them.
There’s a heated race to be the first to use CRISPR safely on humans, that’s for sure. And there’s much pride and benefit that can be gained from being the first to declare success. On the other hand, patients who are hoping to be cured might not really care all that much who gets to be the winner of this all-important race. Ultimately, as long as the treatments prove effective, everybody wins.