CRISPR Treatment Shown to Be Effective Against Aggressive Cancers

Researchers have shown that the CRISPR/Cas9 system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer. The study was published in Science Advances. 

The team from Tel Aviv University developed a novel lipid nanoparticle-based delivery system that specifically targets cancer cells and destroys them by genetic manipulation. The system, called CRISPR-LNPs, carries messenger RNA, which encodes for the CRISPR enzyme Cas9 that acts as molecular scissors that cut the cells' DNA.

"This is the first study in the world to prove that the CRISPR genome editing system can be used to treat cancer effectively in a living animal," said lead researcher Dan Peer. "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. The molecular scissors of Cas9 cut the cancer cell's DNA, thereby neutralizing it and permanently preventing replication."

To examine the feasibility of using the technology to treat cancer, Peer and his team chose two of the deadliest cancers: glioblastoma and metastatic ovarian cancer. 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. Most patients are diagnosed at an advanced stage of the disease when metastases have already spread throughout the body. Despite progress in recent years, only a third of the patients survive this disease. Treatment with CRISPR-LNPs in a metastatic ovarian cancer mice model increased their overall survival rate by 80%.