Researchers at Tel Aviv University (TAU) successfully utilized CRISPR, a gene-editing technology, to remove a single gene from cancer cells in head and neck tumors. The study, conducted in animal models, resulted in the elimination of 50% of the tumors. The findings were published in the journal Advanced Science.
The research was led by Dr. Razan Masarwy, from the lab of Prof. Dan Peer, a global leader in mRNA-based drug development at TAU, according to a press release from the university.
“Head and neck cancers are very common, ranking fifth in cancer mortality,” said Peer. He explained that these are localized cancers that typically begin in the tongue, throat, or neck and can later metastasize.
“If detected early, localized treatment can effectively target the tumor. Our aim was to use the genetic editing of a single gene expressed in this type of cancer to collapse the entire pyramid of the cancerous cell,” he added.
According to the press release, Peer and his team are known for developing mRNA-based drugs encased in synthetic lipid particles that mimic biological membranes. In this study, they designed specialized lipid particles to deliver the CRISPR system in RNA format. These particles were coated with an antibody that targets a protein receptor known as EGF, which is found on cancer cells.
Cutting out the cancer cell's DNA
“These tumors are highly targeted,” explained Peer. “We targeted EGF because the cancer cells express the EGF receptor. Using our nano-lipid delivery system, we injected the drug directly into the tumor in a tumor model 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. Following three injections spaced one week apart, 50% of the cancerous tumors simply disappeared after 84 days - which did not happen in the control group," Peer continued.
In 2020, Peer and his team were the first to use CRISPR to cut genes from cancer cells in mice in a cell-specific manner. This is the first time they have applied it to head and neck cancers.
Peer noted in the press release that CRISPR is not typically used for cancer treatment because researchers previously believed that eliminating a single gene would not be enough to destroy the cancer cells.
However, he explained, “In this study, we demonstrated that there are in fact some genes without which a cancer cell cannot survive, making them excellent targets for CRISPR therapy. Since cancer cells sometimes compensate with other genes, it's possible that additional genes need to be cut out, or perhaps not."
"Theoretically, this approach could be effective against many types of cancer cells, and we are already working on additional cancer types, including myeloma, lymphoma, and liver cancer,” Peer said.