CRISPR-edited immune cells can survive and thrive after infusion into cancer patients

Genetically edited immune cells can persist, thrive, and function months after a cancer patient receives them, according to new data published by researchers from the Abramson Cancer Center. The team showed cells removed from patients and brought back into the lab setting were able to kill cancer months after their original manufacturing and infusion. Further analysis of these cells confirmed they were successfully edited in three specific ways, marking the first-ever sanctioned investigational use of multiple edits to the human genome.

rendering of DNA sequence and cells as part of gene editing from CRISPR cas9

This is the first U.S. clinical trial to test the gene editing approach in humans, and the publication of this new data in Science follows on the initial report last year that researchers were able to use CRISPR/Cas9 technology to successfully edit three cancer patients’ immune cells. Penn is conducting the ongoing study in cooperation with the Parker Institute for Cancer Immunotherapy and Tmunity Therapeutics.

“Our data from the first three patients enrolled in this clinical trial demonstrate two important things that, to our knowledge, no one has ever shown before. First, we can successfully perform multiple edits with precision during manufacturing, with the resulting cells surviving longer in the human body than any previously published data have shown. Second, thus far, these cells have shown a sustained ability to attack and kill tumors,” says senior author Carl June, the Richard W. Vague Professor in Immunotherapy and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center and director of the Parker Institute for Cancer Immunotherapy

The findings are the latest milestone in Penn’s history as cellular and gene therapy pioneers, including development of the first FDA-approved CAR T cell therapy, Kymriah, for pediatric and adult blood cancer patients.

Read more at Penn Medicine News.