Ricerc@Sapienza

NMC is a recently recognized cancer type hallmarked by chromosomal translocation involving the NUT gene, a catastrophic event leading to fusion oncoprotein responsible for malignant transformation and tumor progression. The aggressiveness of disease together with a poor response to conventional treatment make NMC one of the most lethal cancer. Moreover, although until recently NMC has been poorly understood and largely neglected, the number of reported cases is steadily rising.

The antitumour effects of OTX015, a first-in-class BET inhibitor (BETi), were investigated as a single agent or in combination with ionizing radiation (IR) in preclinical in vitro models of rhabdomyosarcoma (RMS), the most common childhood soft tissue sarcoma. Herein, we demonstrated the upregulation of BET Bromodomain gene expression in RMS tumour biopsies and cell lines compared to normal skeletal muscle.