摘要：

Dramatic improvements in the ability to test for all classes of potentially actionable alterations in hundreds or even thousands of genes simultaneously, along with the rapidly expanding portfolio of therapeutic agents targeting a variety of cell signaling networks, have converged to create an unparalleled opportunity to pursue precision medicine in oncology. One obstacle to realizing the full potential of precision medicine in the clinic is the low prevalence of many potentially actionable genomic alterations as well as the diverse range of cancer types potentially implicated. To address this emerging challenge, multi-histology, genomically selected, basket studies have been developed. These studies represent a potentially transformative approach to genomically targeted drug development. In basket studies, patients with tumors known to harbor a qualifying genomic alteration are enrolled irrespective of tumor histology. Tumor types most likely to harbor the genomic biomarker are enrolled to their own cohort. Notably, in these tumor types that enroll to sufficient quantities, efficacy assessments are independently undertaken, therefore accounting for the possibility that tumor lineage may influence drug sensitivity. While the first generation of basket studies were designed mainly to detect early signals of activity across multiple tumor types simultaneously, newer basket studies are now being designed to collect the data required to seek health authority approval. Here we review the current landscape of precision medicine basket studies, with a focus on how to maximize their potential for advancing the standard of care.Citation Format: David Hyman. Basket studies: Lessons learned and future directions. [abstract]. In: Proceedings of the AACR-NCI-EORTC International Conference: Molecular Targets and Cancer Therapeutics; 2015 Nov 5-9; Boston, MA. Philadelphia (PA): AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr PL04-03.

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