Researchers in Israel have found a way to use revolutionary gene-editing technology to treat head and neck cancers.

They say they can identify the single gene that fuels the growth of the cancer cells – and remove it.

The team at Tel Aviv University uses CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a precise and cost-effective way of editing gene sequences to treat genetic disorders, modify immune cells and increase resistance to certain diseases.

CRISPR has, in the last decade or so, been developed to treat numerous conditions, including sickle cell disease, cystic fibrosis, Duchenne muscular dystrophy and HIV.

But the received wisdom was that cancer was too complex for CRISPR.

“Until now, CRISPR wasn’t used for cancer because it was assumed that knocking out a single gene wouldn’t topple the whole structure,” said Prof. Dan Peer, director of the university’s Laboratory of Precision Nanomedicine. “In this study, we demonstrate that some genes are absolutely essential for cancer cell survival, making them excellent targets for CRISPR therapy.”

In 2020, Peer and his team were the first in the world to use CRISPR to cut genes from cancer cells in mice and in a cell-specific manner. This is the first time they have applied it to head and neck cancers.

In experiments with mice, they found that by removing one critical gene, known as cancer-specific SOX2, half the head and neck tumors disappeared completely within 84 days. The mice were given three injections, one week apart.

“These tumors are highly targeted,” said Peer. “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.’ We were happy to observe the domino effect we had predicted.”

The researchers say the same approach could, in theory, be effective against many other types of cancer cells. They are already working on myeloma, lymphoma, and liver cancer.

“Since cancer cells sometimes compensate with other genes, it’s possible that additional genes need to be cut out, or perhaps not,” Peer explained.

Head and neck cancers are the fifth leading cause of cancer-related deaths globally. “These are localized cancers, typically starting in the tongue, throat, or neck, which can later metastasize,” said Peer.

“If detected early, localized treatment can effectively target the tumor. 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. This gene is the cancer-specific SOX2, also expressed in other types of cancer, and overexpressed in these particular tumors.”

The study was led by Dr. Razan Masarwy, from Prof. Peer’s lab. The findings are published in the journal Advanced Science.