First U.S. use of CRISPR to directly target cancer will seek go-ahead from regulators

Sharon Begley:

If all goes as planned, the first clinical trial in the United States testing CRISPR against cancer by altering the DNA of tumor cells inside patients could begin recruiting participants next year, the scientist leading the effort told STAT.

Seventeen studies using CRISPR to treat cancer have been listed on the U.S. registry of clinical trials, but most of those use this genome editing technology to engineer immune cells to attack tumors. That approach, including a pioneering one led by scientists at the University of Pennsylvania, is essentially just a variation on the production of CAR-T cells: CRISPR edits T cells that are isolated from blood that’s been removed from patients, and then the T cells are infused back into the patient. And although researchers in China are rumored to be testing a more direct use of CRISPR against cancer, except for one study using CRISPR to knock out viruses that cause cervical cancer, they have not made details of their plans public.

The Gene Editing Institute at Christiana Care Health System, a nonprofit, private community (as opposed to academic) medical system headquartered in Delaware, is preparing to seek regulatory approval for a much bolder CRISPR cancer study. If it receives the OK from the Food and Drug Administration, which it plans to request in the next few months, it would recruit six to 10 patients with late stage non-small-cell lung cancer and test whether using CRISPR to disable a particular gene would allow standard chemotherapy to work better and longer, ideally buying patients a little more time.