Resumen
High-risk neuroblastoma (NB) is an aggressive cancer of very young children and accounts for almost 15% of all pediatric cancer deaths. Current therapies include high-dose chemotherapy and radiation, which have long-term toxic side effects. Despite these intensive therapies, the overall 5-year survival rate of NB is less than 50%. Therefore, developing novel therapeutic approaches targeting the molecular mechanisms that drive NB progression is very important. In the present study, we repurpose CUDC-907, a dual inhibitor of PI3K and histone deacetylases. These regulators are known to regulate MYCN expression, a key prognostic marker of NB. CUDC-907 potently inhibits NB growth and 3D spheroid tumor growth by inhibiting PI3K, HDAC, and MYCN. Overall, our pre-clinical data demonstrate that repurposing CUDC-907 as a single drug is a novel and effective therapeutic approach for NB.