Ribosomopathies and cancer: pharmacological implications.
Gazmend TemajSarmistha SahaShpend DragushaValon EjupiElisabetta ProfumoElisabetta ProfumoLule BeqaSarmistha SahaPublished in: Expert review of clinical pharmacology (2022)
Despite the association between inherited mutations affecting ribosome biogenesis and cancer biology, the development of therapeutics targeting the essential cellular machinery has only started to emerge. New chemical entities should be designed to modulate different checkpoints (translating oncoproteins, dysregulation of specific ribosome-assembly machinery, ribosomal stress, and rewiring ribosomal functions). Although safe and effective therapies are lacking, consideration should be given to using existing drugs alone or in combination for long-term safety, with known risks for feasibility in clinical trials and synergistic effects.