Scientists have successfully used CRISPR gene-editing technology to eliminate 50% of head and neck tumors in model animals, marking a potential revolution in cancer treatment, Israeli researchers announced on Tuesday. This is the first time CRISPR has been shown to completely eradicate tumors by targeting a single gene — challenging long-held assumptions about the technology’s limitations in cancer therapy.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing technology that allows scientists to precisely alter DNA. It works like molecular scissors, enabling researchers to cut and modify specific genes within an organism’s genome.
It has not been widely used for cancer treatment because it was previously believed that removing a single gene wouldn’t be enough to destroy cancer cells. Tumors often rely on multiple genetic mutations, and cancer cells can compensate when one gene is eliminated. However, the findings by Tel Aviv University scientists demonstrated that some genes, like SOX2, are essential for cancer cell survival, making them viable CRISPR targets. The study also introduced a precise delivery system, overcoming a key obstacle in applying CRISPR to cancer therapy.
The research, led by Tel Aviv University’s Dr. Razan Masarwy, MD, Ph.D., under the guidance of Prof. Dan Peer, was recently published in the peer-reviewed Advanced Science journal.
Head and neck cancers rank fifth in cancer-related mortality and often begin in the tongue, throat, or neck before metastasizing. While current treatments rely on surgery, radiation, and chemotherapy, this pioneering study suggests that precise gene-editing could offer a highly targeted and less invasive alternative.
“Our aim was to use genetic editing of a single gene expressed in this type of cancer to collapse the entire pyramid of the cancerous cell,” said Peer, Director of the university’s Laboratory of Precision Nanomedicine. “This gene is the cancer-specific SOX2, also expressed in other types of cancer and overexpressed in these particular tumors.”
Peer and his team have been at the forefront of developing mRNA-based drugs encapsulated in synthetic lipid particles. In this study, the researchers designed special lipids to carry the CRISPR system in an RNA format. To ensure precision, an antibody targeting the epidermal growth factor (EGF) receptor — a protein commonly expressed in these cancer cells — was attached to the nanoparticles.
“These tumors are highly targeted,” Peer explained. “Using our nano-lipid delivery system, we injected the drug directly into the tumor and successfully took out the gene — literally cutting it out from the cancer cell’s DNA with the CRISPR ‘scissors.’”
The results were striking. Three injections, each spaced one week apart, led to the complete disappearance of 50% of the tumors after 84 days. No similar effect was observed in the control group.
“Generally, CRISPR isn’t used for cancer because the assumption is that knocking out one gene wouldn’t collapse the whole pyramid,” Peer said. “In this study, we demonstrated that some genes are absolutely essential for cancer cell survival, making them excellent targets for CRISPR therapy.”
If this approach proves successful in human trials, it could potentially provide a more precise and less toxic alternative to chemotherapy and radiation.
Encouraged by these findings, the researchers are now working on applying the same method to other cancer types, including myeloma, lymphoma, and liver cancer.
