Abstract

The optimal evaluation of molecularly targeted anticancer agents requires the integration of pharmacodynamic assays into early clinical investigations. Phase '0' trials conducted under the new Exploratory Investigational New Drug Guidance from the US Food and Drug Administration can provide a platform to establish the feasibility of assays for target modulation in human samples, evaluate biomarkers for drug effects and provide pharmacokinetic data. Phase 0 trials could facilitate rational drug selection, identify therapeutic failures early, and might compress timelines for anticancer drug development. We expect that such trials will become a routine part of early-phase oncological drug development in the future.

Author affiliations

1. Robert Kinders, Larry Rubinstein, Ralph E. Parchment, Anthony J. Murgo, Jerry Collins, Oxana Pickeral, Jennifer Low, Sherry Yang, Joseph E. Tomaszewski and James H. Doroshow are at the Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, Maryland, USA.
2. Shivaani Kummar, Seth M. Steinberg, Martin Gutierrez, Lee Helman, Robert Wiltrout and James H. Doroshow are at the Center for Cancer Research, SAIC-Frederick, Inc., NCI-Frederick, Frederick, Maryland USA.

Correspondence to: James H. Doroshow^1,2 Email: doroshoj@mail.nih.gov

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