Researchers at Tel Aviv University have demonstrated that the CRISPR genome editing system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer.

In a paper published this week, the researchers demonstrated a novel lipid nanoparticle-based delivery system that specifically targets cancer cells-and co-author Prof. Dan Peer said it's the first study in the world to prove that the CRISPR/Cas9 can be used to treat cancer effectively in a living animal.

"It must be emphasized that this is not chemotherapy. There are no side effects, and a cancer cell treated in this way will never become active again," said Peer, the VP for R&D and Head of the Laboratory of Precision Nanomedicine at the Shmunis School of Biomedicine and Cancer Research.

"The CRISPR genome editing technology, capable of identifying and altering any genetic segment, has revolutionized our ability to disrupt, repair or even replace genes in a personalized manner."

Peer's team that includes researchers from an Iowa company, Integrated DNA Technologies, and Harvard Medical School, chose two of the deadliest cancers: glioblastoma and metastatic ovarian cancer to examine the system's feasibility. Glioblastoma is the most aggressive type of brain cancer, with a life expectancy of 15 months after diagnosis and a five-year survival rate of only 3%.

The researchers demonstrated that a single treatment with CRISPR-LNPs doubled the average life expectancy of mice with glioblastoma tumors, improving their overall survival rate by about 30%.

Ovarian cancer is a major cause of death among women and the most lethal cancer of the female reproductive system. Despite progress in recent years, only a third of the patients survive this disease-but treatment with CRISPR-LNPs in mice with metastatic ovarian cancer boosted the overall survival rate of by a whopping 80%.

"Despite its extensive use in research, clinical implementation is still in its infancy because an effective delivery system is needed to safely and accurately deliver the CRISPR to its target cells," Peer told Tel Aviv University news. "The delivery system we developed targets the DNA responsible for the cancer cells' survival. This is an innovative treatment for aggressive cancers that have no effective treatments today."

The system, called CRISPR-LNPs, carries a genetic messenger (messenger RNA), which encodes for the CRISPR enzyme Cas9 that acts as molecular scissors that cut the cells' DNA. "The molecular scissors of Cas9 cut the cancer cell's DNA, thereby neutralizing it and permanently preventing replication," explained Peer.

The groundbreaking study was funded by the Israel Cancer Research Fund, and published this week in Science Advances.

The researchers note that by demonstrating its potential in treating two aggressive cancers, the technology opens numerous new possibilities for treating other types of cancer, as well as rare genetic diseases and chronic viral diseases such as AIDS. They intend to proceed with experiments treating Duchenne muscular dystrophy, for instance.

"It will probably take some time before the new treatment can be used in humans, but we are optimistic. The whole scene of molecular drugs that utilize messenger RNA (genetic messengers) is thriving-in fact, most COVID-19 vaccines currently under development are based on this principle," says Peer.

"When we first spoke of treatments with mRNA twelve years ago, people thought it was science fiction. . . We are already negotiating with international corporations and foundations, aiming to bring the benefits of genetic editing to human patients."

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