First CRISPR Trial On Humans
A team of scientists from the Sichuan University’s West China Hospital in Chengdu will begin the first CRISPR human trial for lung cancer patients in August of this year.
Patients who have failed to respond to treatments—who still haven’t gotten well even after undergoing chemotherapy, radiation therapy, and other treatments—were chosen to take part in the CRISPR trial.
Scientists plan to take T cells from patients and use the CRISPR-Cas9 technique to edit out the PD-1 gene. PD-1 regulates T cells’ immune response and prevents them from attacking healthy cells. The team will then multiply the modified samples in the lab before reintroducing them to the patients’ bloodstream, in the hopes that they’ll target parts of the patient’s body affected with cancer.
A biotechnology company will validate the cells before they’re reintroduced into the patient to make sure only PD-1 was edited out.
There are still some researchers who have reservation about the method, like Timothy Chan, an immunotherapy researcher from the Memorial Sloan Kettering Cancer Center in New York City.
Chan said he’s worried the cells may activate an excessive immune response that could target perfectly healthy tissues. He suggests taking T cells directly from a tumor, but the Chinese scientists said the patients’ lung cancer tumors aren’t easily accessible.
As a safety precaution, the Chinese research team will start the trial with just 10 people, and they will slowly administer increasing dosages on just one patient. This is so they can monitor the results closely and to look out for side effects.
Chinese scientists were also the first to use CRISPR editing on human embryos to repair a gene that causes fatal blood disorder, but it only worked on half the embryos.