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Has discovery-based cancer research been a bust?

R J Epstein1

  • 1Department of Oncology, Clinical Cancer Informatics & Research Centre, The Kinghorn Cancer Centre, Sydney, Australia, repstein@stvincents.com.au.

Clinical & Translational Oncology : Official Publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico
|September 5, 2013
PubMed

Insights

High expectations for first-generation cancer diagnostics led to slow clinical progress. Second-generation diagnostics must focus on high-signal data, not just statistical patterns, for better anticancer drug development.

Area of Science:

  • Oncology
  • Genomics
  • Biomarker Discovery

Background:

  • The human genome sequence completion raised hopes for novel anticancer drug development.
  • Clinical progress in oncology has been slower than anticipated over the past decade.

Purpose of the Study:

  • To analyze the impact of first-generation discovery-based diagnostics on anticancer drug development.
  • To propose a framework for more effective second-generation multiplex diagnostics.

Main Methods:

  • Review of clinical progress and diagnostic assay performance in oncology.
  • Analysis of limitations in first-generation multiplex assays (gene expression profiling, proteomics).
  • Identification of key features for second-generation multiplex diagnostics.

Main Results:

  • First-generation multiplex assays have yielded limited high-impact therapeutic targets despite prognostic correlations.
  • Hypothesis-based single-molecule tests show incremental clinical utility.
  • Statistical patterns alone are insufficient for high-impact diagnostic development.

Conclusions:

  • Unrealistically high expectations for first-generation diagnostics have hindered progress.
  • Second-generation multiplex diagnostics require clinically interpretable, high-signal data.
  • Focusing on functional mutations, amplifications, and signaling pathways will advance anticancer drug development.

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