Researchers at the University of Minnesota have completed the first human clinical trial of the CRISPR/Cas9 gene-editing technique, which may help the immune system combat common forms of gastrointestinal (GI) cancer.
Results recently published in The Lancet Oncology show promising signs of the treatment’s safety and potential effectiveness.
In the study, scientists used CRISPR/Cas9 gene-editing technology to modify a type of immune cell known as tumor-infiltrating lymphocytes (TILs). By turning off the CISH gene, the researchers found that the modified TILs were better able to recognize and attack cancer cells.
The treatment was tested on 12 patients with highly metastatic, end-stage cancer and was found to be generally safe, without causing serious side effects from gene editing. Several patients in the study experienced cancer growth arrest, and one patient had complete regression, with metastatic tumors disappearing within a few months and remaining absent for more than two years.
“We believe that CISH is a key factor in preventing T cells from recognizing and destroying tumors,” said study researcher Brendan Moriarty, an associate professor at the University of Minnesota School of Medicine, an investigator at the Masonic Cancer Center, and co-director of the Center for Genome Engineering. “Because it operates inside the cell, it cannot be halted by traditional methods, so we turned to CRISPR-based genetic engineering.”
Unlike other cancer treatments that require fixed doses, this gene editing is permanent and is integrated into T cells from the start.
