Fifteen years ago someone started betting on a new potential anti-cancer target: microRNAs. Now that we know its role much better, the results of the first phase 1 clinical trial of a microRNA inhibitor in oncology are in: microRNA-221, known to promote tumorigenesis. The study is entirely Italian (and academic) and is being carried out by the University of Magna Graecia (UMG) and the Renato Dulbecco University Hospital of Catanzaro thanks to funding from the Airc Foundation. The drug under investigation is called LNA-i-miR-221 and the data, Published on Journal of Hematology and Oncologyindicate that it is ready for experimentation phases 2 and 3 below.

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How the experimental drug LNA-i-miR-221 was born

As the name suggests, microRNAs are small RNA molecules that normally regulate the expression of genes. “We started a decade ago with the idea of ​​identifying and correcting the altered regulation of oncogenic microRNAs,” he explains. Pierfrancesco Tassone, Professor of Medical Oncology at the UMG and coordinator of one of the special programs approved under the AIRC Foundation’s 5 per thousand call for Molecular Clinical Oncology. His research group – together with Pierosandro Tagliaferri, also Professor of Medical Oncology at the UMG – identified microRNA-221 as a possible candidate and therefore designed a specific therapeutic agent. “We have gone through all stages of advanced preclinical development – ​​continues Tassone – following international and national regulatory procedures for drug approval. We have thus obtained a final product that is ready for experimental clinical use in humans for the first time.” It is therefore a new frontier in the field of oncology (there has only been one clinical trial with microRNA inhibitors in humans with the hepatitis C virus, the researchers say).

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I study

Phase 1 studies represent the first step in the clinical development of a drug: They are carried out on a small number of patients and are not intended to prove the effectiveness of a new drug, but to investigate its pharmacokinetics and the maximum tolerated dose. A total of 19 patients with different types of advanced solid tumors (breast, colon, stomach, liver, ovaries, pancreas, glioblastoma and mesothelioma) enrolled between February 2019 and December 2021 participated in this first study of LNA-i-miR-221 . Of these, 17 – 11 women and 6 men, with an average age of over 60 years, were suitable for the analysis over a mean observation period of 56 days. The results demonstrated the safety and biological activity of this first microRNA-221 inhibitor.

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An independent translational research

“It is the realization of an ambitious project”, comments Tassone, “implemented exclusively with the support of AIRC and its supporters, testifying to the possibility of completing an independent path of translational research, starting from an idea and from the laboratory and a Drug developed.” by making it available in pharmaceutical formulation to get to the treatment of patients. A path that in our country is mainly industrial and rarely academic and independent, especially in the phases of development and characterization of the new drug.” The researchers hope that the research of this world-class drug will stimulate the international community to invest human and financial resources to invest in the development of new therapy scenarios. “We are proud to have contributed to this important milestone of Professor Pierfrancesco Tassone and his group, which AIRC has continuously supported for over fifteen years,” he concludes Federico Caligaris Cappio, Scientific Director of the AIRC Foundation – We recognize that research cannot be improvised but is built over time with accuracy and method. It is therefore important to be able to offer the best scientists financial stability for long-term projects.”

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