Emerging New Therapeutics for Retinoblastoma

Background: Over the last few decades, chemotherapy has become the main treatment of retinoblastoma, delivered through various routes: intravenous (IVC), intra-arterial (IAC), and intravitreal (IvitC). Despite its efficacy, chemotherapy related toxicity (ocular and systemic) and recurrences due to resistant tumor clones are common, highlighting the need for novel therapeutic agents. Summary: Recent advances in our understanding of the molecular drivers of Rb1 tumorigenesis and mechanisms of tumor resistance have afforded opportunities to explore novel targets such as the MDMX–p53 pathway (Nutlin-3), Histone deacetylase inhibitors (HDACi), Spleen tyrosine kinase (SYK) inhibi tors, and genetic and immune modulatory drugs. In this review we discuss the limitations of current therapeutic strategies, candidate cellular pathways, current evidence for newer targeted drugs, and offer a look towards the future. Key messages: Advances in the understanding of the molecular drive rs of RB pathway have provided opportunities to explore novel drugs with targeted effects, improved bioavailability, and reduced chemotoxicity.
Source: Ocular Oncology and Pathology - Category: Opthalmology Source Type: research