Scientists Report First use of CRISPR to Substitute Genes to Treat Patients with Cancer

For the first time, scientists have used CRISPR gene editing technology to substitute a gene to treat patients with cancer. The new approach first identifies T cells that specifically recognize and kill an individual patient's tumor cells.

T cells are a type of immune cell, and the cells in question kill tumor cells when they encounter specific genetic mutations from cancer. However, there are often not enough of these cells. By CRISPR editing other T cells from the patient with the same cancer recognition motifs, the approach is designed to build a large army of cancer-fighting cells, which are then infused back into the patient, thus greatly amplifying that natural anti-tumor response.

The therapy approach was developed by PACT Pharma, based upon the work pioneered by Dr. Jim Heath, president of the Institute for Systems Biology (ISB), and his collaborators, UCLA Professor Dr. Toni Ribas and Caltech Professor Dr. David Baltimore. The therapy was used to treat 16 patients. The early-stage clinical trials were meant to show that the treatment was safe for patients, but the researchers also found that the engineered T cells did, in fact, home in on the patient tumors.

“This is a leap forward in developing a personalized treatment for cancer,” Ribas said. “The generation of a personalized cell treatment for cancer would not have been feasible without the newly developed ability to use the CRISPR technique to replace the immune receptors in clinical-grade cell preparations in a single step,” he added.

To learn more about this research, read the article on the ISB website, or watch this YouTube video.