Researchers at University of Minnesota have completed the first human clinical trial using CRISPR/Cas9 gene-editing technology aimed at helping the immune system fight advanced forms of gastrointestinal (GI) cancer.
Recently published in The Lancet Oncology, the study reports encouraging signs of safety and potential effectiveness, marking an important step forward in cancer immunotherapy research.
In the trial, scientists used CRISPR/Cas9 gene editing to modify a specific type of immune cell known as tumor-infiltrating lymphocytes (TILs). By disabling the CISH gene, researchers found that the edited T cells were better able to recognize and attack cancer cells. The CISH gene is believed to suppress immune responses inside the cell, making it a key obstacle in effective tumor destruction.
The experimental treatment was tested on 12 patients with advanced, highly metastatic, end-stage cancers. Results showed that the therapy was generally safe, with no serious side effects directly linked to the gene-editing process.
Notably, several patients experienced stabilization of tumor growth, and in one remarkable case, metastatic tumors completely regressed and remained absent for more than two years following treatment.
“We believe CISH is a major internal checkpoint that limits the ability of T cells to recognize and destroy tumors,” said study investigator Brendan Moriarty, associate professor at the University of Minnesota Medical School and researcher at the Masonic Cancer Center. “Because it operates inside the cell, it could not be blocked using conventional drugs, which is why we turned to CRISPR-based genetic engineering.”
Unlike many traditional cancer treatments that require repeated dosing, this gene-editing approach creates a permanent modification within the patient’s T cells from the start. Researchers emphasize that while these findings are promising, larger clinical trials are needed to fully assess long-term effectiveness and broader applicability.
This breakthrough highlights the growing potential of precision gene editing as a powerful tool in next-generation cancer therapies.
