Clinical Research Papers:

Comprehensive tumor profiling identifies numerous biomarkers of drug response in cancers of unknown primary site: Analysis of 1806 cases

Zoran Gatalica _, Sherri Z. Millis, Semir Vranic, Ryan Bender, Gargi D. Basu, Andreas Voss and Daniel D. Von Hoff

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Oncotarget. 2014; 5:12440-12447. https://doi.org/10.18632/oncotarget.2574

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Zoran Gatalica1, Sherri Z. Millis1, Semir Vranic2, Ryan Bender1, Gargi D. Basu1, Andreas Voss1, Daniel D. Von Hoff3

1Caris Life Sciences, Phoenix, United States of America

2Department of Pathology, Clinical Center, University of Sarajevo, Sarajevo, Bosnia and Herzegovina

3Translational Genomic Research Institute and Virginia G Piper Cancer Center, Phoenix, United States of America

Correspondence to:

Sherri Millis, e-mail: [email protected]

Keywords: cancer of unknown primary, multiplatform, molecular profiling, mutations, targeted therapy

Received: September 04, 2014     Accepted: October 03, 2014     Published: October 31, 2014


Background: Cancer of unknown primary (CUP) accounts for approximately 3% of all malignancies. Despite extensive laboratory and imaging efforts, the primary site usually cannot be unequivocally confirmed, and the treatment for the most part remains empirical. Recently, identification of common cancer pathway alterations in diverse cancer lineages has offered an opportunity to provide targeted therapies for patients with CUP, irrespective of the primary site.

Patients and Methods: 1806 cancers of unknown primary were identified among more than 63,000 cases profiled at Caris Life Sciences. Multiplatform profiling of the tumor samples included immunohistochemistry, gene sequencing and in situ hybridization methods in an effort to identify changes in biomarkers that are predictive of drug responses.

Results: Biomarkers associated with a potential drug benefit were identified in 96% of cases. Biomarkers identified included those associated with potential benefit in nearly all classes of approved cancer drugs (cytotoxic, hormonal, targeted biological drugs). Additionally, biomarkers associated with a potential lack of benefit were identified in numerous cases, which could further refine the management of patients with CUP.

Conclusion: Comprehensive biomarker profiling of CUP may provide additional choices in treatment of patients with these difficult to treat malignancies.

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