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Human Biospecimens: A Critical Resource for Translational Research and Molecular Medicine


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Title: Human Biospecimens: A Critical Resource for Translational Research and Molecular Medicine

  • Human Biospecimens A Critical Resource for
    Translational Research and Molecular Medicine
  • Update on the Development of the National
    Biospecimen Network

Carolyn Compton, MD, PhD Director of
Biorepositories and Biospecimen Research National
Cancer Institute
Data Are Becoming Available on an Unprecedented
Molecular Data
Biorepositories Support Discovery in
Translational Research and Molecular Medicine
Biorepositories in the United States
  • More than 300 million specimens are stored from
    gt150 million cases, and gt20 million new specimens
    are collected each year.
  • We still dont have what we need.
  • Shortage of high-quality specimens has been
    identified repeatedly as a major barrier to
    progress in translational research.
  • Technologically advanced approaches that can
    greatly accelerate translational research depend
    for their performance and development on
    uniformly collected, processed, annotated and
    stored biological samples.

Handbook of Human Tissue Sources, RAND 1999
NCI Biorepository Report Rand
  • gt50 M invested annually by NCI in
    biorepository-related programs
  • 125 programs collect, maintain and/or store
    approximately 4 million biospecimens per year
  • The programs support research at all levels
    basic, epidemiologic, translational and clinical
  • Most collect frozen biospecimens and support
    genomic and proteomic research
  • NCI-supported programs do not employ common SOPs
    or QA/QC measures
  • No common database for these programs exists nor
    is there a defined mechanism to access

NCI Biorepository Report
  • NCI-supported biorepositories are not
    coordinated to optimize resource value.

Maximizing Biospecimen Quality
  • Existing Challenges for Biorepositories
  • Scarce and variable clinical data
  • Variable biospecimen collection, processing, and
    storage methods
  • Varying informatics infrastructures and data
    elements laborious exchange of information and
  • Varying approaches to patient consent and privacy
  • Lack of standardization at all levels

NBN Concept Developed with Extensive Input from
the Cancer Community
  • RAND Report on Case Studies of Existing
    Biorepositories Best Practices for a
    Biospecimen Resource in the Genomic and
    Proteomic Era
  • (http//
  • National Biospecimen Network Blueprint
  • (http//

Key Requirements for a New Biorepository System
The National Biospecimen Network (NBN)
  • Diversity of cancer types and populations based
    on continual review of researcher needs
  • Access through a timely, centralized peer-review
  • Ethical and privacy compliance through a chain of
  • Resources provided without intellectual property
  • Best practice- and data-driven based SOPs to
    enable reproducible and comparable results
  • Pathology and clinical annotation (including
  • State-of-the-art informatics system to streamline
    the research production process and create in
    silico research capability
  • Communication and outreach efforts

Prostate SPOREs NBN Pilot Project
NCIs Specialized Programs of Research Excellence
  • Program established in 1992
  • Now 58 SPOREs focusing on 14 organ sites
  • Funded by P50 grants
  • Conduct interdisciplinary, translational research
  • Each SPORE has an existing tissue bank
  • The 11 prostate SPOREs agree to pilot the NBN

Prostate SPORE Programs and Principal
Designated by Prostate SPORE PIs as primary
points of contact for the NBN pilot
Goals for Testing the NBN Concept with the
Inter-Prostate SPORE Biomarkers Study (IPBS)
  • Determine the feasibility of establishing a
    biorepository network based on the NBN concept
  • Evaluate a controlled and harmonized approach to
    biospecimen collection, processing, storage,
    dissemination and clinical annotation for a
    specific scientific study
  • Pilot an infrastructure to integrate specimen
    banks to enhance the quality and availability of
    specimens and associated data
  • All of the above with an eye towards generalizing
    the concept for the broader scientific community

Prospective Biomarkers Study Goals
  • Conduct a multi-institutional Inter-Prostate
    SPORE prospective validation study of five
    promising prognostic biomarkers selected by
  • Conduct limited, focused retrospective studies of
    three promising biomarkers that require
    additional evidence of prognostic utility before
    conducting prospective validation studies
  • Establish a resource of well-characterized tissue
    and serum samples linked to clinical and
    epidemiological data for future biomarker
    discovery and validation

A Pilot Framework for the NBN Concept
  • Collect high-quality biospecimens with detailed
    annotation suitable for biomarker validation
  • Employ standardized protocols for biospecimen
    collection, annotation, processing, storage, and
  • Create an informatics infrastructure that links
    multiple programs and allows exchange of data and
  • Develop common policies and procedures to govern
    prioritization and access to specimens stored in
    a common repository
  • Establish a process to verify and validate the
    quality of biospecimens to ensure that they are
    suitable for high throughput analyses of the
    genome, transcriptome, and/or proteome

Model for Patient Enrollment and Sample Collection
  • SPORE Sites
  • SPORE Sites

Prostate SPORE NBN Pilot Informatics Vision
Prostate SPORE NBN Pilot Informatics Vision,
Meanwhile Back at the NCI
  • Report to the National Cancer Advisory Board An
    Analysis of NCI-Supported Programs that Collect,
    Store, and Distribute Human Biospecimens for
    Research Purposes
  • Challenge by the NCAB
  • What have we already got?
  • What is the quality?
  • What is it good for?

Biospecimen Coordinating Committee (BCC) is born
  • The BCC
  • Representatives from all NCI divisions
  • Review all existing authoritative literature and
    policy on biobanking
  • Establish SOP recommendations and guidelines for
    NCI-funded biorepositories
  • Establish an evaluation system for existing

Premise of BCC Activities
  • Premise biospecimen quality utility for
    research is dependent on
  • The physical quality of the biologic samples
  • The quality, amount and type of associated
    clinical data
  • Appropriate consent
  • Use is limited by ethical, legal, and policy
  • Harmonized banking practices based on scientific
    data (where available) or best practices will
    improve the quality of NCI-funded biobanks and
    the quality of the research based on these

The Biospecimen Coordinating Committee
  • Two workshops to inform these recommendations
  • Biospecimen Access and Ethical, Legal, and Policy
    Issues (the ELP Workshop)
  • Arthur Caplan, University of Pennsylvania, Chair
  • June 23-24, 2005
  • Best Practices and Recommendations for
    Establishing and Maintaining Biorepositories That
    Support Cancer Research (the Biospecimen
    Collection Workshop)
  • Mark Rubin, Dana-Farber Cancer Institute, Chair,
  • July 18-20, 2005

The NBN Ahead?
  • There are no national standards for tissue
    repositories that collect and store specimens for
    research use.
  • Standards still remain to be defined.
  • The work of the NBN Pilot Project and the BCC
    will inform the NBN plan
  • The NCI Biorepository Report, Rand

Lack of Standardization Undermines Discovery
Rapid Increase in Microarray Publications
Results Vary
Map of discordance. An experiment at NIH found
that three commercial devices rated different
genes as being turned on (red) and turned off
(green) in a single batch of pancreatic cells
Source SCIENCE, 22 OCT 2004, Getting in the
Noise Out of Gene Arrays
New Thinking for Biobanking
  • The Stone Age didnt end because they ran out of