Gene therapy that induces the body to create microRNA-22 (miR-22), a naturally occurring molecule, successfully treated mice with hepatocellular carcinoma, the most common form of liver cancer.
The miR-22 treatment also reduced liver inflammation and produced better survival outcomes with no observable toxicity compared to the FDA-approved liver cancer treatment lenvatinib.
Those are the findings of a new study from the UC Davis Comprehensive Cancer Center published in Molecular Therapy.
“This research introduces miR-22 gene therapy as a promising and innovative approach for treating hepatocellular carcinoma,” said Yu-Jui Yvonne Wan, senior author of the study. Wan is a distinguished professor and vice chair for research in the UC Davis Department of Pathology and Laboratory Medicine.
“The study’s findings suggest that miR-22 therapy could provide better survival outcomes, enhance anti-tumor immunity, improve metabolism and reduce inflammation.”
The University of California filed a patent application for Wan’s discovery of miR-22 for treating hepatic and metabolic diseases. The patent is currently pending.
MicroRNAs’ role in health and disease
MicroRNAs are small molecules that contain ribonucleic acid (RNA), a type of genetic material. MicroRNAs are widely found in plants and animals. They are “non-coding,” meaning they do not make proteins like some other RNA molecules.
Using an inactivated adenovirus, they introduced miR-22 into the mice with a single intravenous injection.
The mice treated with the gene therapy were compared with mice treated with the current FDA-approved drug lenvatinib (administered orally once a day), untreated mice and healthy mice.
Both miR-22 and lenvatinib inhibited the progression of the liver cancer compared to untreated mice. However, the miR-22-treated mice had longer survival times without toxicity compared with lenvatinib-treated mice.
