Download NCI Resources for the Development of Early Stage Cancer

NCI Resources for the Development of
Early Stage Cancer Technologies
Mark D. Lim, Ph.D.
[email protected]
Montreal In Vivo 2010
June 8, 2010
US National Cancer Institute
Stimulating innovation by reducing risk
Discovery and Validation: Access to high-quality biospecimens
Office of Biorepositories and Biospecimen Research
Early stage technology development
Program for Innovative Molecular Analysis Technologies
What is a biospecimen?
Window into the health of an individual
Identification of targets for drug development, treatment and prevention
Identify biologic variations that determine drug efficacy and drug toxicity
Defining markers for susceptibility, screening and reoccurrence
Validation of new therapeutics and diagnostics
Blood is a complex biospecimen
• Travels through all tissues in the body
• 60,000 miles of vessels
• Important for tumor growth and metastasis
• Readily collected in the clinic
Numerous Roles:
• delivery of nutrients
• removal of metabolites
• delivery of oxygen
• removal of carbon
dioxide
• immune response
• response to injury
• etc…
Complex Composition:
Biologically active!
• red blood cells
• white blood cells
• platelets
• almost every protein
• genomic transcripts
• etc…
Office of Biorepositories and
Biospecimen Research
Tissue Variables:
 Antibiotics
 Other drugs
 Type of anesthesia
 Duration of anesthesia
 Arterial clamp time
How
wetemperature
tell biologic
 Timecan
at room
 Temperature of room
 Type of fixative
 Time in fixative
 Rate of freezing
 Size of aliquots
Blood Variables:
• Patient Health, Hydration, Diet
• Medications
• Patient Position
• Tourniquet time duration
• Gauge of needle
• Order
of draw
fact
from
biologic artifact?
• Type of tube, additives
• Serum vs plasma vs whole blood
• Processing Procedure
• Processing Temperature
• Centrifuge Speed
• Centrifuge Temperature
• Storage Temperature
• Freeze-thaw
Office of Biorepositories and
Biospecimen Research
The historical focus of NCI funding
Patient
Medical/
Surgical
Procedures
Acquisition
Handling/
Processing
Storage
Distribution
Scientific
Analysis
8. Biobanks
By ALICE PARK
Inside Huntsman
Cancer Institute's
vaults: Pancreatic
tumors on ice.
Lance W. Clayton
for TIME
Folks at the National Cancer Institute (NCI) are
heading up an effort to establish the U.S.'s first national
biobank — a safe house for tissue samples, tumor
cells, DNA and, yes, even blood — that would be used
for research into new treatments for diseases…. By
fall, the group hopes to have mapped out a plan for a
national biobank; the recent stimulus showered on the
government by the Obama Administration might even
accelerate that timetable.
March 12, 2009
Restocking
Unused
Sample
NCI Best Practices
• Evidence-based, data-driven technical and
operational standards to ensure quality and enable
reproducible molecular analysis
• High-quality biospecimen annotation with pathological
and clinical data
• Biospecimen access through a timely, centralized peer
review process
• Ethical and privacy compliance through a chain of
trust with research participants
“Living document”
updated
to stay current with
state-of-the-science
and policies
• State-of-the-art informatics systems to track
biospecimens, associated data, and research participant
informed consents
• Communication and outreach efforts, particularly
with investigators, to ensure greatest impact
http://biospecimens.cancer.gov
An International Effort
Standardisation and improvement of generic pre-analytical tools
and procedures for in-vitro diagnostics
• 4 year project from EU’s FP7 Programme (9 M Euros)
• 16 partners from 11 different European Countries
• 7 private research companies (ex: QIAGEN, Dako, Aros, etc.)
• 8 public research organizations (ex: Technical University Munich, IARC)
• Europrean Standards Organization (CEN)
• Trials
1) SPIDIA-DNA - evaluation of DNA quality/quantity from whole blood sample
2) SPIDIA-DNAplas – evaluation of DNA quality/quantity from whole blood sample
plus the evaluation of DNA quality/quantity/integrity from plasma sample
3) SPIDIA-RNA – evaluation of RNA quality/quantity/stability from whoel blood
Sample
http://www.spidia.eu
Biobanks
KAUST Biobank (Saudi Arabia)
National; Disease-based; to support biomedical research
Estonian Genome Project
National; Population-based
UK Biobank
National; Population-based; Aged 45-69
GenomEUtwin (Finland)
International; Population-based; Twin cohorts
BBMRI
Pan-European; Network of existing and de novo biobanks
(population, twin, and clinical/control cases)
Biobank Japan
National; Hospital-patient based recruitment; Focused mainly on common diseases and
supporting pharmacogenetics research
Singapore Tissue Network
National; Tissue and DNA Bank to facilitate translational and population research for
Singapore; Collects, processes, and disseminates tissue samples for specific research
projects
caHUB Principles
•
Standardized biospecimen collection and
distribution procedures
•
Standardized data sets and data vocabulary
•
Harmonized approached to ethical and legal
issues
– Standardized consent, MTAs
•
Transparent governance and business models
– Transparent access policies
•
Large well-designed specimen sets for a
variety of research questions
How can caHUB serve your needs?
…dialogue is just beginning
Working with NIST – standards for pharma and biotech
- biomarker discovery/validation efforts based on stored blood samples
Trans-institute
Trans-agency
International
PPP
GTEx
NIST
SPIDIA
Patient
Advocacy
NIH Office of
Rare Diseases
NCI IMAT Program
Mission:
Revolutionize the state-of-the-science by stimulating the early-stage
development of next generation molecular and cellular analysis technologies
Goals:
•
Innovation - Support out-of-the-box approaches for analyzing cancer
•
Interdisciplinary - Support highly innovative technology development projects
from all communities
•
Proof-of-concept - Support general innovative technology approaches and
prototype development (application)
http://innovation.cancer.gov
Unique Attributes of IMAT
•
Emphasis on high-risk, high-impact, and high-payoff technology development
•
Investigator-initiated to identify unmet need and provide solution
•
Emphasis on technology development (vs. traditional hypothesis-driven)
•
Milestone-based, with performance milestones that quantitatively address
measures such as specificity, sensitivity, speed, and similar performance
parameters
•
Staged process requiring quantitative evidence of progress or feasibility before
advancement to the next stage
•
All communities (industry/academic, international) are invited to apply
•
Review process focused on improvement over state of the art - approach
IMAT Themes
“Innovative Technology Development” RFA-CA-10-005
Modified R21: 3 years and $500K in direct costs
No preliminary data required
Quantitative milestones are required
“Emerging Technology Development”
Early stage development- R21: 2 years and $275K in direct costs
No preliminary data required - Quantitative milestones are required
RFA-CA-10-003
Advanced development- R33: 3 years and no budget cap
Proof of concept data required
RFA-CA-10-004
IMAT Themes
“Innovative Technology Development” RFA-CA-10-005
Modified R21: 3 years and $500K in direct costs
No preliminary data required
Quantitative milestones are required
“Emerging Technology Development in Biospecimen Sciences”
Early stage development- R21: 2 years and $275K in direct costs
No preliminary data required - Quantitative milestones are required
RFA-CA-10-001
Advanced development- R33: 3 years and no budget cap
Proof of concept data required
RFA-CA-10-002
U.S. Small business opportunities
NCI SBIR Development Center - http://sbir.cancer.gov/
PHASE I – R41, R43
– Feasibility Study
– $100K and 6-month (SBIR) *
– or 12-month (STTR) Award
PHASE II – R42, R44
• Full Research/R&D
• $750K and 2-year Award (SBIR & STTR) *
• Commercialization plan required
Phase II Bridge Award
PHASE III
• Commercialization Stage
• Use of non-SBIR/STTR Funds
* Note: Actual funding levels may differ by topic.
Non-traditional/Interdisciplinary
approaches
David Beebe, Ph.D. (Engineering - Univ. Wisc. Madison)
R21 Microfluidic Channels for High Density, High Performance Culture Assays
Gregory Faris, Ph.D. (Applied Physics - SRI International)
R21 Signatures for Functional Optical Imaging of Cancer
Stuart Lindsay, Ph.D. (Biophysics - Arizona State Univ)
R21 Mapping Epigenetic Modifications at the Nanoscale: Aptamers for
Microscopy
David Nolte, Ph.D. (Physics - Purdue)
R21 Highly Multiplexed Assays on the BioCD for Acute Lymphocytic Leukemia
Supported projects in the market
Jonathan D. Oliner, Ph.D., Affymetrix
Reverse-Engineering Signal Transduction Networks (R43 - 1998)
Gary Latham, Ph.D., Ambion, Inc. (RNA Later)
Enzymatic Tools for Degrading Tissue and Preserving RNA
(R43 - 2001; R44, 2005-2007)
Robert H. Daniels, Ph.D., Quantum Dot Corp. (Invitrogen)
Sensitive, Multiplexed Analysis of Breast Cancer Markers
(R44 - 1999)
Darren Link, Ph.D., Raindance Technologies
Exon Specific Sequencing of Whole Genomic DNA (R21 –2007)
Supporting Canadian Innovation
University of Toronto
Shana O. Kelly, PhD
Development of DNA-templated IR quantum dots
Scott Tanner, PhD
Development of (Prototype) Bead Array Flow Cytometer
with Mass Spectrometer Detection
Univ. of Toronto Spinout Rolls Out New Biomarker
System with Eye on Bead Array Market
Genome Web – May 21, 2010
Industry-based innovation
Implementation of innovative RNA sample quality control methods
Collaboration between BioTrove (Life Technologies)
and University of Toledo
High-throughput oncogene mutation detection in human cancer
Based on Sequenom Technology
Releasable Antibodies for Multiplexed Analysis of Cancer
Biomarkers
Transformative HTS cell migration assay for rapid screening of
cancer therapeutic
Advanced Technology for Assaying Cancer-Drug Resistance
miRNA Profiling in Fixed Cancer Samples
Now part of Applied Biosystems
Other NCI Opportunities
Check websites or contact program official for updated information
Division of Cancer Biology
Integrated Cancer Biology Program (ICBP)
Tumor Microenvironment Network (TMEN)
PAR-09-026
Division of Cancer Control and Population Sciences
Methods and Technologies Branch
Division of Cancer Prevention
Early Detection Research Network (EDRN)
Division of Cancer Treatment and Diagnosis
Cancer Imaging Program (http://imaging.cancer.gov)
Exploratory Studies in Cancer Detection, Diagnosis and Prognosis (R21, PA-08-267)
Developmental Research in Cancer Prognosis and Prediction (R21,R33, PA-09-158)
Exfoliated Cells and Circulating DNA in Cancer Detection and Diagnosis (R21, PA-09-238)
Avenues for
Public-Private-Partnerships
1. Identify a scientific or clinical problem better answered in partnership than alone!
2. Partnership Options:
* Established directly between the NIH (as a whole or through one or more
ICs) and one or more outside entities.
* Developed via the Foundation for NIH (FNIH).
* Involve one or more other charitable foundations.
3. Discuss how to structure the relationships to optimize the science.
4. Memorialize the PPP goals and structure in a MOU and clear it appropriately.
Rules for NIH PPP






Science driven
Rigorous
Fair
Inclusive
Compliant with Federal law, regulation and policy
Priority to the agency
http://ppp.od.nih.gov/
Biomarkers Consortium
• Facilitate the development and validation of biomarkers using
new and existing technologies
• Help qualify these biomarkers for specific applications in
diagnosing disease, predicting therapeutic response, or
improving clinical practice
• Generate information useful to inform regulatory decisionmaking
• Make consortium project results broadly available to the entire
scientific community
www.biomarkersconsortium.org
Websites
NCI Office of Biorepositories and Biospecimen Research (OBBR)
http://biospecimens.cancer.gov
NCI Program for Innovative Molecular Analysis Technologies (IMAT)
http://innovation.cancer.gov
NIH Public Private Partnerships Office
http://ppp.od.nih.gov/
Additional questions?
[email protected]