Abstract 3221: Development of patient-derived xenograft (PDX) models for gastrointestinal stromal tumors (GIST) as a pre-clinical platform for drug development

Abstract

Abstract Patients with gastrointestinal stromal tumors (GIST) continue to face a poor prognosis. GISTs are a type of soft tissue sarcoma primarily affecting adults over the age of 50. They are the most common mesenchymal tumor within the GI tract. Survival rates vary greatly depending on the location when it is first diagnosed and there has been minimal progress in improving the survival rates of metastatic GISTs. It has been difficult to accurately evaluate the effectiveness of targeted drugs to develop treatment strategies due to the rarity and complexity of GISTs. In an effort to overcome this challenge, we have established PDX models to recapitulate the original patient tumor. In this work, we selected four primary GIST samples based on mutational and clinical profile. PDX models were developed in immunocompromised mice and were further characterized by immunohistochemistry, sequencing and pharmacological efficacy. We then evaluated the efficacy of chemotherapeutic agents in these models. Our data demonstrates that these preclinical PDX models could be used in evaluation and development of novel drug candidates for GIST. Citation Format: Jill Ricono, Chelsea Mullins, Praveen Nair, Cyrus Mirsaidi, Thomas Broudy. Development of patient-derived xenograft (PDX) models for gastrointestinal stromal tumors (GIST) as a pre-clinical platform for drug development. [abstract]. In: Proceedings of the 106th Annual Meeting of the American Association for Cancer Research; 2015 Apr 18-22; Philadelphia, PA. Philadelphia (PA): AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 3221. doi:10.1158/1538-7445.AM2015-3221