The first human test in the U.S. involving the gene-editing tool CRISPR could begin at any time and will employ the DNA cutting technique in a bid to battle deadly cancers.
Doctors at the University of Pennsylvania say they will use CRISPR to modify human immune cells so that they become expert cancer killers, according to plans posted this week to a directory of ongoing clinical trials.
The study will enroll up to 18 patients fighting three different types of cancer-multiple myeloma, sarcoma, and melanoma-in what could become the first medical use of CRISPR outside China, where similar studies have been under way.
The CRISPR trial, led by doctor Edward Stadtmauer, involves reprogramming a person’s immune cells to find and attack tumors. To help enhance the treatment, Penn scientists intend to use CRISPR to delete two genes in patients’ T cells to make them better cancer fighters. In the study, doctors will remove people’s blood cells, modify them with CRISPR in the lab, and then infuse them back into the patients.
CRISPR Therapeutics, a biotech company based in Cambridge, Massachusetts, asked European regulatory authorities in December for permission to try to cure beta thalassemia, a blood disorder, by making a genetic tweak to people’s blood cells.