A smart therapy to “switch off” tumors

Reducing the growth of tumors through RNA molecules that can control genetic alterations in diseased cells and correct their defects: this is the result of an international study in which Italy also participated with three research institutes of the National Research Council of Italy


Reducing the growth of tumors with RNA molecules capable of controlling genetic alterations in diseased cells and correcting their defects: this is the result of an international study in which researchers from the National Research Council of Italy (CNR), and the University of Eastern Piedmont, in collaboration with colleagues from prestigious American institutions, participated: The Cancer Research Institute of Beth Israel Deaconess Medical Center and Harvard Medical School in Boston, the Department of Cancer Biology and Molecular Medicine of the Beckman Research Institute of the City of Hope Medical Center (Duarte, California) and the University of Singapore.

The results of the research are published in Nature Communications: the study intervenes in the process known as DNA methylation, the epigenetic mechanism that “silences” genes, preventing their activity, which has been shown to have a direct correlation with the development of cancer.

This process is governed by specific proteins, named DNA methyltransferases, the control of which can provide new promising therapeutic strategies to counteract methylation abnormalities in tumors: this is what the researchers of the group, coordinated by Annalisa Di Ruscio (BIDMC-HMS ), Carla Lucia Esposito (Cnr-Ieos), Vittorio de Franciscis (Cnr-Irgb), and Marcin Kortylewski (COH) have achieved by generating RNA molecules that can effectively and selectively block the DNA methylation process, and thus reactivate the genes erroneously silenced in the disease.

“Until now, the only compounds capable of blocking this process of genetic alteration in cancer cells were highly toxic and non-selective”, says Vittorio de Franciscis (Cnr- Irgb). “Our technique, on the other hand, allows to act in a targeted manner, opening a new therapeutic frontier in the so-called precision medicine: it uses small RNA molecules (AptaDiR) which act selectively by blocking a specific DNA methyltransferase and thus promoting a significant reduction of tumor growth”.

The results, derived from the integration of the different expertise of the involved research groups, pave the way for a new class of intelligent RNA therapies, safe and effective, which would revolutionize current oncological therapies by improving not only the clinical outcome, but also the quality of life of cancer patients.

“Moreover, this targeted approach could also be extended to other diseases, marking a fundamental turning point in the treatment of incurable genetic diseases resulting from methylation defects”.

For Information:
Vittorio de Franciscis
Cnr – Irgb




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