Recent advances in molecular profiling have shown that cancers arising from the ovary are phenotypically and genetically heterogeneous. Within histologies, many mutations in druggable targets are uncommon in frequency but mutations leading to activation of specific signal transduction pathways are common. These results support the notion that different targeted agents should be prioritized for testing between and within ovarian cancer histologies. The subsegmentation of ovarian cancers based on molecular features challenge traditional trial designs. Feasibility of accrual and need for data on biological and clinical consequence or target inhibition are leading to trial designs that lump or split patient populations by histology, pathway, gene, and/or mutation. This review summarizes potential therapeutic targets identified from recent molecular profiling studies of ovarian cancers and trial designs to evaluate targeted agents in rare cancer settings.
Keywords: genomics; ovarian cancer; targeted drugs; trial designs.