Scientists from Dr. Jingfang Ju's research group in the Department of Pathology at Stony Brook University have engineered a powerful dual-action molecule that successfully destroys treatment-resistant non-small cell lung cancer (NSCLC). NSCLC frequently develops resistance to standard chemotherapies and targeted drugs like EGFR inhibitors. This occurs because tumor cells mutate and adapt. The innovative therapy, named Gem-miR-129, combines a natural cancer-fighting tumor suppressor RNA molecule with a built-in chemotherapy drug to eliminate tumors from within. The newly developed Gem-miR-120 simultaneously inhibits three key oncogenic-driver proteins (HMGB1, YAP1, and PBX3) that cancer cells use to survive and resist treatment. Unlike traditional genetic therapies, Gem-miR-129 does not require a complex delivery vehicle. It enters cancer cells entirely on its own. It deploys miR-129 to halt cancer growth while releasing the hidden chemotherapy drug gemcitabine. It reduces tumor-infiltrating T-regulatory cells, allowing the body's immune system to fight back. The drug destroys cells that are already resistant to major therapies such as erlotinib and osimertinib. The therapy shrank tumors in mice with NSCLC and significantly prolonged survival. Crucially, the subjects experienced no noticeable toxic side effects. Researchers state this successful proof-of-concept paves the way for future Investigational New Drug (IND) enabling studies in human patients.
An article featuring this advancement has been published in BioWorld and can be found here.