Microlin Bio, Inc., a development stage biotechnology company focused on the development of microRNA based therapeutics for cancer, has announced that results from a study demonstrating the potential of its lead compound, AntimiR-21 (AM-21) to be an effective therapy for lung cancer were published in the journal Molecular Pharmaceutics.
MiR-21 is a microRNA mainly involved in regulation of gene expression. Overexpression of miR-21 has been linked to progression of non-small cell lung carcinoma (NSCLC) and other cancers. AM-21 therapy along with lipid nanoparticles (LNP) inhibit miR-21 activity, resulting in upregulation of tumor suppressor genes that are targets of miR-21.
The study, entitled “Lipid Nanoparticles Composed of Quaternary Amine-Tertiary Amine Cationic Lipid Combination (QTsome) for Therapeutic Delivery of AntimiR-21 for Lung Cancer,” was led by Dr. Robert Lee of The Ohio State University. It utilized AM-21 encapsulated in the QTsome LNPs (QT/AM-21) to increase the efficacy and improve the delivery of AM-21 to tumor cells. A combination of QTsome encapsulated AM-21 and Paclitaxel (PTX) was also evaluated against lung carcinoma cells.
In vitro, QT/AM-21 displayed high potency against A549 cells (lung carcinoma cells) and increased their sensitivity to PTX. Treatment with QT/AM-21 also led to upregulation of target genes such as PTEN and DDAH1 and reduced metastatic potential of the lung carcinoma cells.
In vivo, QT/AM-21 treatment prolonged survival in the A549 mouse xenograft tumor model by modulating targets of miR-21. Similar to the in vitro results, QT/AM-21 increased the sensitivity of the tumor cells to PTX in vivo.
Joseph Hernandez, Chief Executive Officer and Executive Chairman of Microlin Bio, commented, “Results from this study highlight AM-21′s potential as a highly effective treatment strategy for lung cancer. Our findings show that AM-21′s spectrum of anti-tumor activity could be very effective against NSCLC and other types of cancers. Data from this study also suggest that this therapy maybe valuable to patients who have developed resistance to paclitaxel. The combination of QT/AM-21 and paclitaxel demonstrated a reduction in cell proliferation and increased therapeutic activity. Overall, the results are highly encouraging and validate our strategy for further evaluation of AM-21 as an antitumor agent. We look forward to initiating additional studies to assess the safety and efficacy of QT/AM-21.”
MicroRNAs are recently discovered naturally occurring RNA molecules (composed of 19 to 25 nucleotides) that do not encode proteins but instead regulate gene expression and various biological pathways. The improper balance of microRNAs is linked to many diseases, including cancer. As such, replacement or inhibition of deregulated microRNAs may act as a potent means to treat cancers.