Abstract 6895: Application of MiniPDX® in clinical indication identification and anti-tumor drug development

Abstract MiniPDX® (Mini-patient-derived xenograft) is a novel, rapid (7~10days), and accurate method for drug sensitivity testing in vivo. It was referred as in vivo version of organoid assy. The testing material can be fresh patient tumor samples or tissues from established PDX models. We have systematically evaluated and compared the response rates of MiniPDX® assays and PDX assays pairwise in 26 PDX models across 3 types of cancers to 12 clinically relevant regimens for chemical and targeting drugs. The results demonstrate a high correlation between drug responses of the two assays, with sensitivity of 80% and specificity of 93%. More and more studies showed that MiniPDX® drug sensitivity test results were consistent with clinical responses in most patients, which indicated MiniPDX® models have great potential in guiding personalized medicine. LIDE has successfully conducted over 3, 000 MiniPDX® tests for clinical precision medicine, covering more than 50 indications. The application of MiniPDX® extends beyond therapeutic selection, facilitating the prioritization of approved pharmacological interventions and informing the discovery pipeline for novel agents. Importantly, the MiniPDX® Mouse Trial using fresh tumor samples generated from the clinic is beneficial for determination of potential clinical indications. Only thousands of fresh cells leftover from MiniPDX® preparation are sufficient to get genomic and transcription data by OncoVee ® K-cell technology. The combination of MiniPDX® assay and omics data would be very useful for determination of potential biomarkers to distinguish responders and non-responders in population with certain indication (s) and leveraged for patient stratification and selection criteria in clinical trial design. In addition, we have developed a new version of MiniPDX® assay for immunotherapy (IO-FIVE, Immuno-Oncology Fast In Vivo Efficacy test) by using fresh patient cancer cells with autologous immune cells to maximally mimic human tumor microenvironment before seeding in immune-deficient mice. This assay allows for the tracking of cellular viability and phenotype pre- and post-treatment, thus providing a robust metric of in vivo immunotherapeutic efficacy within a fortnight. Citation Format: Bin Xie, Hongkui Chen, Shizhu Zhao, Le Li, Song Xi, Yu Di, Yang Liu, Xinlei Chen, Xiaorong Gu, Zhenle Bi, Kaimeng Hu, Loc Van, Josh Caggiula, Candice Tang, Danyi Wen. Application of MiniPDX® in clinical indication identification and anti-tumor drug development abstract. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts) ; 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84 (6Suppl): Abstract nr 6895.