Mirna Therapeutics, Inc. and Horizon Discovery Ltd. announced today the publication of new data providing further confirmation that microRNA-34 (miR-34), and the tumor suppressor pathways it controls, is an important potential target for a wide variety of cancers. The data are published in Molecular Therapy, the official journal of the American Society of Gene and Cell Therapy, and reveals a novel mechanism through which miR-34 exerts its tumor suppressor function in cancers.
Mirna is currently conducting a Phase I study of MRX34, a miR-34-based therapy and the first microRNA mimic to advance into a human clinical trial. The Phase I study focuses on patients with unresectable primary liver cancer or metastatic cancer with liver involvement.
The data published in the paper entitled: "TP53-independent function of miR-34a via HDAC1 and p21CIP1/WAF1" demonstrate that the tumor suppressive activity of miR-34 is the same in p53-positive and p53-negative cancer cells. The study further revealed the existence of a p53-independent pathway by which miR-34 induces the expression of p21, a potent tumor suppressor inhibiting uncontrolled cellular proliferation. The endogenous level of p21 was shown to be critical for maximal miR-34 activity.
The research was supported by a grant from the National Institutes of Health, and a commercialization grant from the Cancer Prevention and Research Institute of Texas (CPRIT).