MicroRNAs (miRNAs) are a type of noncoding RNA (RNA that do not codify for proteins but have regulatory functions) that has been shown to be differentially expressed in numerous tissues, key cellular processes and multiple diseases.  Medical communities have significantly underestimated the spectrum of consequences that their altered expression may cause in patients.  In cancer, miRNAs act by targeting crucial tumor suppressor proteins in a post-transcriptional manner.  During the past years, they have been proven to be involved in the processes of tumor initiation, progression and metastases; moreover, their signatures have been associated with diagnosis, staging, progression, prognosis and response to treatment.  Since therapeutic strategies based on the modulation of miRNA activity have not met the needed requirements, we are in the urgent need of exploiting new treatment options.  We recently embarked on the development of small molecules that target oncogenic miRNAs (rather than proteins) for cancer therapy. We named these agents SMIRs (small molecule inhibitors targeting miRNAs), and are focusing on using these small molecules to target overexpressed miRNAs as a new therapeutic approach.  By performing high-throughput screening cellular-based assays, we are aiming to achieve a transformative concept that can be extended to multiple oncogenic miRNAs as well as different cancer types, bringing these molecules to the clinical practice.

Targeting microRNAs with small molecules: A novel therapeutic approach for cancer treatment

247th National Meeting (2014)

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