biostudies-literatureSrimongkol AMahidol UniversityThailand Research FundHealth System Research Institute39https://www.ebi.ac.uk/biostudies/studies/S-EPMC9890748biostudies-literatureUnknown42(1)<h4>Background</h4>Recurrence of retinoblastoma (RB) following chemoreduction is common and is often managed with local (intra-arterial/intravitreal) chemotherapy. However, some tumors are resistant to even local administration of maximum feasible drug dosages, or effective tumor control and globe preservation may be achieved at the cost of vision loss due to drug-induced retinal toxicity. The aim of this study was to identify drugs with improved antitumor activity and more favorable retinal toxicity profiles via screening of potentially repurposable FDA-approved drugs in patient-derived tumor organoids.<h4>Methods</h4>Genomic profiling of five RB organoids and the corresponding parental tissues was performed. RB organoids were screened with 133 FDA-approved drugs, and candidate drugs wereJournal of experimental & clinical cancer research : CRSunitinib efficacy with minimal toxicity in patient-derived retinoblastoma organoids.PMC9890748136/2562MRG628008363-027Kaewkhaw RRojanaporn DHongeng SSrimongkol AChaitankar VThanomchard TLaosillapacharoen NSaengwimol DBorwornpinyo SfalseSunitinib efficacy with minimal toxicity in patient-derived retinoblastoma organoids.<h4>Background</h4>Recurrence of retinoblastoma (RB) following chemoreduction is common and is often managed with local (intra-arterial/intravitreal) chemotherapy. However, some tumors are resistant to even local administration of maximum feasible drug dosages, or effective tumor control and globe preservation may be achieved at the cost of vision loss due to drug-induced retinal toxicity. The aim of this study was to identify drugs with improved antitumor activity and more favorable retinal toxicity profiles via screening of potentially repurposable FDA-approved drugs in patient-derived tumor organoids.<h4>Methods</h4>Genomic profiling of five RB organoids and the corresponding parental tissues was performed. RB organoids were screened with 133 FDA-approved drugs, and candidate drugs were2023-01-01T00:00:00Z2023 Feb2026-07-16T21:13:24.182Z2025-04-05T18:52:37.208ZS-EPMC98907483672611010.1186/s13046-023-02608-1