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Abstract 2968: Organoid based functional genomics approach to identify treatment options for patients with rare cancers

Rare cancers present a distinct challenge for the identification of effective therapeutic strategies. While these tumors make up 20% of total cancer cases in the United States, they are categorically defined as occurring in less than 6 of every 100,000 people, limiting the utility of traditional methods to study, and identify treatments for these cancers. Though there have been tremendous advances in cancer therapeutics, identification of treatments for rare tumors have lagged, leaving patients with rare tumors with worse 5 year survival rates than individuals diagnosed with common cancer types. The goal of precision oncology is to identify effective therapeutic strategies for each individual patient. With high throughput sequencing it is now possible to identify the genetic events that give rise to and sustain each individual tumor in clinically relevant time frames, however our capacity to leverage this information to identify effective therapeutic options remains limited. This gap in our capacity stems from a lack of clinically relevant approaches that would allow physicians to accurately gauge the impact of multiple therapeutic strategies and relate them to clinical responses. Through the establishment of an iterative Functional Genomics Pipeline which integrates the development and analysis of high-fidelity patient derived organoid models of rare cancers with personalized drug sensitivity testing, and comprehensive genomics our work suggests that personalized modeling could have clinical utility for the identification of treatment options for individual patients with rare cancers. Citation Format: Benjamin David Hopkins, Julie-Ann Cavallo-Fleming, Sally Claridge, Lamberto De Boni, Chantal Pauli. Organoid based functional genomics approach to identify treatment options for patients with rare cancers [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2968.