Clinical Development of TargomiRs, a miRNA Mimic-Based Treatment for Patients with Recurrent Thoracic Cancer

Abstract

miRNAs are responsible for post-transcriptional control of gene expression, and are frequently downregulated in cancer. It has become well established that restoring miRNA levels can inhibit tumor growth, and many studies have demonstrated this in preclinical models. This in turn has led to the first clinical trials of miRNA replacement therapy. This special report focuses on the development of TargomiRs – miRNA mimics delivered by targeted bacterial minicells – and the very first clinical experience of a miRNA replacement therapy in thoracic cancer patients in the Phase I MesomiR-1 trial.

Keywords::

• EDVs
• malignant pleural mesothelioma
• mesomiR-1
• miRNA
• miR-16
• targeted delivery

Financial and competing interests disclosure

Research in the authors’ laboratories is supported by a Translational Program Grant from the Cancer Institute NSW (11TPG3–06 to N van Zandwijk, G Reid, SC Kao and N Pavlakis) and the Cancer Council NSW (RG14–17 to G Reid, N van Zandwijk, J MacDiarmid and H Brahmbhatt). G Reid and N van Zandwijk are holders of a patent covering consensus mimics. J MacDiarmid and H Brahmbhatt are Co-Directors and shareholders in EnGeneIC Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Additional information

Funding

Research in the authors’ laboratories is supported by a Translational Program Grant from the Cancer Institute NSW (11TPG3–06 to N van Zandwijk, G Reid, SC Kao and N Pavlakis) and the Cancer Council NSW (RG14–17 to G Reid, N van Zandwijk, J MacDiarmid and H Brahmbhatt). G Reid and N van Zandwijk are holders of a patent covering consensus mimics. J MacDiarmid and H Brahmbhatt are Co-Directors and shareholders in EnGeneIC Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. No writing assistance was utilized in the production of this manuscript.