Abstract IA02: The Treehouse Childhood Cancer Initiative

Abstract

Abstract Each year over 4,000 children with cancer in the US lack adequate treatment options for their disease, underscoring the urgent need for new therapeutic targets and new therapies. Cancer genomic investigations focusing on the identification of recurrent DNA changes in tumors that could be targeted by therapies have been disappointing in the majority of pediatric cancers, as these diseases have lower rates of somatic mutation compared to adult cancers. To address this challenge, the UC Santa Cruz Genomics Institute launched the Treehouse Childhood Cancer Initiative, an innovative program that identifies novel therapeutic targets for difficult-to-treat pediatric cancers through leveraging the power of large, shared tumor RNA-sequencing datasets. Treehouse partners with pediatric cancer hospitals, including Lucille Packard Children’s Hospital at Stanford, UCSF Benioff Children’s Hospital, Children’s Hospital of Orange County, Nationwide Children’s Hospital, and British Columbia Children’s Hospital, to investigate whether the comparative RNA-seq approach is feasible and useful when applied to patients prospectively. A preliminary analysis of 126 patients from four hospitals showed that comparative RNA-seq analysis can identify druggable targets for over 74.3% of difficult-to-treat pediatric cancer patients, while state-of-the-art DNA analysis finds potential therapeutic options for only 45.9% of patients. Current work focuses on determining the clinical utility of the Treehouse approach. Citation Format: David Haussler. The Treehouse Childhood Cancer Initiative [abstract]. In: Proceedings of the AACR Special Conference on Advancing Precision Medicine Drug Development: Incorporation of Real-World Data and Other Novel Strategies; Jan 9-12, 2020; San Diego, CA. Philadelphia (PA): AACR; Clin Cancer Res 2020;26(12_Suppl_1):Abstract nr IA02.