Co-Clinical Trial: An Innovative Platform for Drug Development

Abstract

Co-clinical trials, as defined by the National Cancer Institute (NCI), encompass parallel or sequential trials of combination therapy conducted in both patients and in mouse models, including human-in-mouse models with relevant genotypes mirroring those of the patients under study. This approach has evolved primarily for treating cancers with diverse genetic mutations to enable targeted therapy. Cancer, being a multifaceted and highly heterogeneous disease characterized by numerous genetic abnormalities and disruptions in interconnected signalling pathways, presents significant hurdles in developing effective cancer treatments. In practice, only a small fraction, approximately 5%, of anti-cancer drugs that undergo preclinical testing ultimately secure approval from the FDA. Ongoing efforts aim to address the challenges associated with translating research findings, particularly by integrating mouse models into personalized medicine, leading to the inception of “Co-clinical trials.” The Co-Clinical Trial Project focuses on utilizing genetically engineered mouse models (GEMMs) or patient-derived xenograft (PDX) models to guide patient treatment strategies in concurrent human clinical trials and aid in selecting suitable chemotherapy agents. These models are cultivated in specialized “Mouse hospitals,” and to streamline the integration process, the “Co-clinical imaging resource program” has been introduced to translate insights from animal models to clinical trials. This review aims to provide a comprehensive overview of the components of co-clinical trials and the challenges encountered in this approach.

Keywords: Co-clinical trial; Mouse hospital; Patient derived xenograft;