First #CRISPR clinical trial gets green light from US panel : Nature News & Comment
CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials.
On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR–Cas9 to help augment #cancer therapies that rely on enlisting a patient’s T cells, a type of immune cell.
“Cell therapies [for cancer] are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. Gene editing could improve such treatments and eliminate some of their vulnerabilities to cancer and the body’s immune system, he says.
The researchers will remove T cells from 18 patients with several types of cancers and perform three CRISPR edits on them. One edit will insert a gene for a protein engineered to detect cancer cells and instruct the T cells to target them, and a second edit removes a natural T-cell protein that could interfere with this process. The third is defensive: it will remove the gene for a protein that identifies the T cells as immune cells and prevent the cancer cells from disabling them. The researchers will then infuse the edited cells back into the patient.