Utilizing Existing Clinical and Population Biospecimen Resources for Discovery or Validation of Markers for Early Cancer Detection

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• Overview
• Purpose
• Agenda
• Workshop Summary
• Planning Committee
• Related Resources
• Key Publications

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Leah E. Mechanic, PhD, MPH
Program Director, Genomic Epidemiology Branch
mechanil@mail.nih.gov

Overview

Despite the large investment in biomarker discovery and validation, very few major cancer biomarkers have been approved for clinical use over the last 25 years. In addition, many initially promising biomarkers have not been validated. One major problem is that certain study designs result in specimens that are not ideal for biomarker discovery or validation. For example, if there is a systematic difference between cases and controls or in handling of their specimens, a biased comparison occurs.

To accurately discover and/or validate cancer biomarkers, access to resources where serial collections, epidemiological data, clinical data, and outcomes were assessed over time is required. Specimens collected just-proximal to the diagnosis of cancer are ideal because they are typically not biased by knowledge of the diagnosis or treatment of the disease. These include serial blood samples (including plasma or serum) collected in a systematic and equivalent manner, with appropriate epidemiological and clinical annotation; such samples are ideal for discovery and/or validation of biomarkers for early detection.

There are several NCI- and NIH-sponsored cohort studies and clinical trials with high-quality, well-annotated specimens that have been utilized to assess biomarker validity. Important characteristics of these cohorts and trials needed to perform such assessments are the availability of serial blood draws and the collection of outcomes data (e.g., development of cancer) over time. However, much larger numbers and disease-free populations are needed. Consequently, it may be useful to aggressively leverage existing clinical and epidemiology cohort resources since new collections are so expensive and time-consuming.

Purpose

The aim of the meeting was to learn whether ongoing NCI-funded projects, e.g. cohort studies, health maintenance organizations (HMOs), or clinical trials, might be leveraged for unbiased studies of biomarker study and validation. Cancer researchers from these diverse studies met to discuss the challenges and opportunities for using existing infrastructures. The workshop had three main goals:

1. Facilitate sharing of methods, approaches, and lessons learned in performing this type of research using existing clinical and epidemiology infrastructures, mainly for studies of cancer early detection.
2. Identify obstacles or challenges that need to be addressed to perform this type of research.
3. Develop best practices and contrasting alternative approaches to be documented in a white paper.

Agenda

Workshop Summary

Schully SD, Carrick DM, Mechanic LE, Srivastava S, Anderson GL, Baron JA, Berg CD, Cullen J, Diamandis EP, Doria-Rose VP, Goddard KAB, Hankinson SE, Kushi LH, Larson EB, McShane LM, Schilsky RL, Shak S, Skates SJ, Urban N, Kramer BS, Khoury MJ, Ransohoff DF. Leveraging biospecimen resources for discovery or validation of markers for early cancer detection. JNCI. 2015; 107(4) [Epub ahead of print].

Planning Committee

• Muin Khoury, MD, PhD, EGRP, Division of Cancer Control and Population Sciences (DCCPS), NCI
• Barry Kramer, MD, MPH, Division of Cancer Prevention (DCP), NCI
• Leah Mechanic, PhD, MPH, EGRP, DCCPS, NCI
• David F. Ransohoff, MD, University of North Carolina at Chapel Hill
• Sheri Schully, PhD, EGRP, DCCPS, NCI
• Sudhir Srivastava, PhD, MPH, Cancer Biomarkers Research Group (CBRG), DCP, NCI