title: 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.
authors.affiliation: Asbestos Diseases Research Institute, Concord, NSW 2139, Australia.|Sydney Medical School, The University of Sydney, Camperdown, NSW 2006, Australia.
authors.forename: Glen
authors.initials: G
authors.lastname: Reid
authors.affiliation: Asbestos Diseases Research Institute, Concord, NSW 2139, Australia.|Sydney Medical School, The University of Sydney, Camperdown, NSW 2006, Australia.|Department of Medical Oncology, Chris O'Brien Lifehouse, Camperdown, NSW 2050, Sydney, Australia.
authors.forename: Steven C
authors.initials: SC
authors.lastname: Kao
authors.affiliation: Kolling Institute of Medical Research, University of Sydney, St Leonards, NSW 2065, Australia.|Department of Medical Oncology, Royal North Shore Hospital, St Leonards, NSW 2065, Australia.
authors.forename: Nick
authors.initials: N
authors.lastname: Pavlakis
authors.affiliation: EnGeneIC Ltd, Sydney, Australia.
authors.forename: Himanshu
authors.initials: H
authors.lastname: Brahmbhatt
authors.affiliation: EnGeneIC Ltd, Sydney, Australia.
authors.forename: Jennifer
authors.initials: J
authors.lastname: MacDiarmid
authors.affiliation: Kolling Institute of Medical Research, University of Sydney, St Leonards, NSW 2065, Australia.|Department of Medical Oncology, Royal North Shore Hospital, St Leonards, NSW 2065, Australia.
authors.forename: Stephen
authors.initials: S
authors.lastname: Clarke
authors.affiliation: Sydney Medical School, The University of Sydney, Camperdown, NSW 2006, Australia.|Department of Medical Oncology, Chris O'Brien Lifehouse, Camperdown, NSW 2050, Sydney, Australia.
authors.forename: Michael
authors.initials: M
authors.lastname: Boyer
authors.affiliation: Asbestos Diseases Research Institute, Concord, NSW 2139, Australia.|Sydney Medical School, The University of Sydney, Camperdown, NSW 2006, Australia.
authors.forename: Nico
authors.initials: N
authors.lastname: van Zandwijk
mesh_terms: D017321:Clinical Trials, Phase I as Topic; D006801:Humans; D008175:Lung Neoplasms / Q000628:therapy*; D008654:Mesothelioma / Q000628:therapy*; D000086002:Mesothelioma, Malignant; D000067492:RNAi Therapeutics / Q000379:methods*; D013899:Thoracic Neoplasms / Q000628:therapy*
keywords: EDVs; malignant pleural mesothelioma; mesomiR-1; miR-16; miRNA; targeted delivery