CI4CC Moonshot Blue Ribbon Panel Report 20161010
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Transcript of CI4CC Moonshot Blue Ribbon Panel Report 20161010
Blue Ribbon Panel Report 2016
CI4CC in UtahOctober 10 th, 2016
www.cancer.gov/brp
Warren A. Kibbe, [email protected]
@wakibbe
NCI and The Cancer Moonshot
Town HallOctober 5, 2016
Douglas R. LowyActing Director, National Cancer Institute,
National Institutes of Health
3
An Exciting Time for Cancer Research
Commitment of current Administration Precision Medicine Initiative
(PMI®) in oncology
Cancer Moonshot Strong, bipartisan Congressional
support for cancer research
4
Relationship Between Bypass Budget and Blue Ribbon Panel Report Bypass Budget addresses NCI’s
entire research portfolio
Lays out the plan for NCI’s continued investment in cancer research
Cancer Moonshot is a unique opportunity to enhance cancer research in specific areas that are poised for acceleration
The BRP report made 10 bold, yet feasible, recommendations that will fast-track initiatives if infused with Moonshot funding
Continuing to strongly support investigator-initiated research
Changes in RPG pool: FY12-FY16 and beyond
7
Focus on specific cancers with health disparities
Some examples: lung cancer, colorectal cancer, liver cancer, breast cancer, prostate cancer, multiple myeloma
Identify the risk factors and their relative contribution to the disparities: biologic factors, life-style factors, health care access/utilization
Explore efforts to mitigate the risk factors
■ White
♦ Black▲ API
* AI/AN
● Hispanic*
*Hispanic is not mutually exclusive from other groupsIncidence data from SEER 13 1992-2010, Mortality data from NCHS
8*Hispanic is not mutually exclusive from other groupsIncidence data from SEER 13 1992–2013, Mortality data from NCHS
Colon & Rectum
1992
1995
1998
2001
2004
2007
2010
2013
0
10
20
30
40
50
60
70
80 SEER Incidence
Year of diagnosis
Inci
denc
e ra
te p
er 1
00,0
00
1992
1995
1998
2001
2004
2007
2010
2013
0
10
20
30
40
50
60
70
80 US Mortality
Year of deathM
orta
lity
rate
per
100
,000
9
“…Mutations in a set of 15…genes appear to be strongly preferentially associated with CRCs arising in AA versus Caucasian individuals, suggesting an important difference in the mutational landscapes of CRCs arising in different ethnic groups. “
Guda et al., 2015. Proc. Natl. Acad. Sci. 112:1149
■ White
♦ Black▲ API
* AI/AN
● Hispanic*
*Hispanic is not mutually exclusive from other groupsIncidence data from SEER 13 1992-2010, Mortality data from NCHS
10*Hispanic is not mutually exclusive from other groupsIncidence data from SEER 13 1992–2013, Mortality data from NCHS
Myeloma
1992
1995
1998
2001
2004
2007
2010
2013
0
3
6
9
12
15 SEER Incidence
Year of diagnosis
Inci
denc
e ra
te p
er 1
00,0
00
1992
1995
1998
2001
2004
2007
2010
2013
0
3
6
9
12
15 US Mortality
Year of deathM
orta
lity
rate
per
100
,000
11
Some Principles to Follow
Develop better genomic, biologic, environmental, and treatment response information about cancer in minority populations
Minority populations represented in clinical trials & preclinical cancer models
Ensure from the beginning that appropriate minority representation will be included
Two new NCI research initiatives Early onset malignancy initiative: the first
minority-based cancer tissue bank; early onset tumors; collect information on treatment, response, and outcome Detailed molecular characterization of fully
annotated tumors Organized through NCORP (NCI Community
Oncology Research Program) Develop new cancer models from tumors of
minority patients
13
Goals of the Cancer Moonshot
Accelerate progress in cancer, including prevention & screening From cutting edge basic research to wider uptake of standard of
care
Encourage greater cooperation and collaboration Within and between academia, government, and private sector
Enhance data sharing(Presidential Memo 2016)
14
Cancer Moonshot Federal Task Force
Vice President’s Office
“Blue Ribbon Panel”
Working Groups
National Cancer Advisory Board
NCI/NIH
Vice President’s Cancer Moonshot Workflow
15
Cancer Moonshot: Why now?
The science is ready, and would benefit from a major infusion of additional resources
Lots of opportunities for bold, but feasible, initiatives that could have important implications for our understanding of cancer and for patients through improved prevention, screening, and treatment
Immunotherapy has come of age
16
Unintentional communication that cancer is now a technological/engineering problem? Terms such as “precision medicine”: do they
inadvertently imply understanding that is greater than it is, and that advances in cancer no longer depend on scientific discovery of the unknown?
Immune checkpoint inhibitors: based on understanding immune regulation, but still much that we don’t understand
Emphasize: progress in cancer remains heavily dependent on developing new knowledge
17
An Opportunity for Focused Research to Accelerate Progress Take advantage of current advances in the
understanding of cancer and recent technological innovation
Apply the knowledge and innovation to focus on specific projects that can have a substantial impact on understanding and/or improvement for patients
NB: NCI will continue to support a great deal of other meritorious research
cancer.gov/brp Blue Ribbon Panel Report 2016
Blue Ribbon Panel
Recommendations focus on what we believe can be accelerated now by Cancer Moonshot funding
Recommendations are limited – they do not touch the entire spectrum of what NCI supports!
The recommendations do not include implementation!
The BRP recommendations have identified opportunities that the community thinks are important. NCI plans to implement them. But, the rate and extent are dependent on the level of appropriations we receive!
In the near term, there will be a call for NCI scientific staff to get engaged to help design programs and design implementation by participating in working groups.
Co-ChairsTyler Jacks* Elizabeth Jaffee*
Dinah Singer MIT Johns Hopkins
NCIPeter C. Adamson, M.D.*Children's Hospital of Philadelphia
James AllisonMD Anderson
David AronsNational Brain Tumor Society
Mary BeckerleUniv. of Utah
Mitchel Berger*UCSF
Jeffrey BluestoneParker Institute
Chi Dang*U. Penn
Mikael DolstenPfizer
*NCAB/BSA member
Augusto OchoaLouisiana State Univ.
Jennifer PietenpolVanderbilt Univ.
Angel PizzaroAmazon Web Services
Barbara RimerUNC
Charles Sawyers*MSK
Ellen SigalFriends of Cancer Research
Patrick Soon-ShiongNantWorks
Wai-Kwan Alfred YungMD Anderson
James DowningSt. Jude Hospital
Levi GarrawayHarvard Medical School
Gad GetzBroad Institute
Laurie GlimcherWeill Cornell
Lifang HouNorthwestern
Neal KassellUniv. Va.
Elena Martinez*UCSD
Deborah MayerUNC
Edith MitchellThomas Jefferson Univ.
Blue Ribbon Panel
cancer.gov/brp Blue Ribbon Panel Report 2016
Charge to Blue Ribbon Panel
“The Blue Ribbon Panel … will provide expert advice on the vision, proposed scientific goals, and implementation of the National Cancer Moonshot. ..The panel may also recommend other cancer research activities to enhance this effort.
“The Panel will provide an intensive examination of the opportunities and impediments in cancer research… the Panel may call upon special consultants, assemble ad hoc work groups … Findings and recommendations of the Panel will be reported to the NCAB.
(Presidential Memo 2016)
Working Group Co-Chair NCI Staff
Cancer Immunology Liz Jaffee,Jim Allison
Toby Hecht, Kevin Howcroft
Precision Prevention and Early Detection
Mary Bekerle,Jennifer Pietenpol
Elisa WoodhouseTracy Lively
Tumor Evolution Chi Dang, Levi Garraway
Joanna Watson, Suresh Mohla, Tony Dickherber
Clinical Trials Charles Sawyers,Mitch Berger
Jeff HildesheimMeg Mooney
Implementation Sciences Elena Martinez,Augusto Ochoa
Bob Croyle, Worta McCaskill-Stevens
Pediatric Cancer Peter AdamsonJim Downing
Judy MietzMalcolm Smith
Enhanced Data Sharing Angel PizarroGaddy Getz
Juli KlemmBetsy Hsu, Jennifer Couch
BRP Working Groups
cancer.gov/brp Blue Ribbon Panel Report 2016
Blue Ribbon Panel Working Groups
• The seven Working Groups had 12-15 members.
• In total almost 150 individuals were engaged in the Working Groups, including academic researchers, clinicians, industry representatives and advocates.
• Charge was to generate 2-3 recommendations of major scientific opportunities that are poised for acceleration.
• The Working Groups met almost weekly to discuss and formulate their recommendations
cancer.gov/brp Blue Ribbon Panel Report 2016
Scientific and Community Outreach ActivitiesGoal:
Provide opportunities for the public and experts ways to submit ideas
Increase the public’s participation in the Cancer Moonshot
Approaches:
Online public idea repository
One-on-one public input: email
BRP Listening sessions
Professional conferences
Response:
Over 1600 ideas received from the public!
cancer.gov/brp Blue Ribbon Panel Report 2016
Charge to Blue Ribbon Panel
“The Blue Ribbon Panel … will provide expert advice on the vision, proposed scientific goals, and implementation of the National Cancer Moonshot. ..The panel may also recommend other cancer research activities to enhance this effort.
“The Panel will provide an intensive examination of the opportunities and impediments in cancer research… the Panel may call upon special consultants, assemble ad hoc work groups … Findings and recommendations of the Panel will be reported to the NCAB.
(Presidential Memo 2016)
cancer.gov/brp Blue Ribbon Panel Report 2016
The 7 Working Groups submitted a total of 14 recommendations
All 14 were discussed at the July 20 meeting of the Blue Ribbon Panel
Thirteen were approved as “Moonshot recommendations”
One recommendation was converted to a demonstration project
Other recommendations across working groups were combined to form the 10 final recommendations in the report
Overview of Blue Ribbon Panel Report
cancer.gov/brp Blue Ribbon Panel Report 2016
In the final recommendations, the following recommendations were combined:
Pediatrics Cancer and Tumor Evolution Working Groups both recommended efforts to identify new therapeutic targets to overcome cancer resistance.
Tumor Evolution, Cancer Immunology and Precision Prevention Working Groups proposed generation of human atlases of cancer.
Both the Precision Prevention and Implementation Science Working Groups focused on the importance of screening.
Development of technologies cited throughout the recommendations was combined into a single recommendation
Overview of Blue Ribbon Panel Report
cancer.gov/brp Blue Ribbon Panel Report 2016
The Report summarizes these recommendations of exceptional research opportunities that could lead to powerful advances in our understanding of cancer
Three sidebars highlight proposed demonstration projects
The online Report includes all recommendations in their entirety at https://cancer.gov/brp
Overview of Blue Ribbon Panel Report (Cont.)
cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the RecommendationsA. Network for direct patient engagement:
• Enlist patients in federated network that includes patient tumor profiling data and “pre-registers” patients for clinical trials.
B. Cancer immunotherapy translational science network.
• Organize a network to discover and evaluate novel immune-based approaches for adult and pediatric cancers, and eventually develop vaccines.
C. Therapeutic target identification to overcome drug resistance.
• Launch interdisciplinary studies to delineate mechanisms that lead cancer cells to become resistant to previously effective treatments.
D. Creation of a national cancer data ecosystem.
• Create an ecosystem to collect, share, and interconnect datasets.
cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the RecommendationsE. Fusion oncoproteins in pediatric cancer.
• Improve understanding of the abnormal fusion proteins that result from chromosomal translocations and drive many pediatric cancers.
F. Symptom management research.
• Support research to accelerate development of guidelines for management of patient-reported symptoms to improve quality of life and adherence to treatment regimens.
G. Precision prevention and early detection:
• Implementation of evidence-based approaches. Conduct implementation science research to encourage broader adoption of HPV vaccination, colorectal cancer screening, and tobacco cessation.
cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the Recommendations (continued)H. Retrospective analysis of biospecimens from patients treated with standard
of care.
• Analyze biopsies to learn which features predict outcome to better plan treatment for future patients.
I. Creation of human tumor atlas.
• Catalog genetic lesions and cellular interactions in tumor/immune/other cells in tumor microenvironment.
J. Development of new enabling technologies.
• Support development of technologies to accelerate testing of therapies and tumor characterization.
cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the Demonstration ProjectsPrevention: Lynch Syndrome Demonstration Project
• A national effort to systematically screen all CRC and endometrial cancer patients for Lynch syndrome (LS)
• First degree relatives of patients with LS would be given the option to be screened and provided with genetic counseling
Therapy: Pediatric Cancer Immunotherapy Network Demonstration Project• A national pediatric immunotherapy clinical trials network to facilitate the
testing of new immunotherapy approaches in childhood cancer• Establish a robust research pipeline to advance pediatric immunotherapy
Emergent Technologies: Tumor Pharmacotyping Demonstration Project• Develop intra- and extra-tumoral technologies for determining the most
effective therapeutic agents for individual patients
cancer.gov/brp Blue Ribbon Panel Report 2016
National network of patient biological and clinical data
Prevention
Health disparities research
Biomarkers
Development of technology and preclinical models
Data sharing, analytics and predictive computational modeling
Collaboration; public-private partnerships
Cross-Cutting Themes
cancer.gov/brp Blue Ribbon Panel Report 2016
Cross-Cutting Themes (Cont.)
Improving the lives of patients is the major cross-cutting theme of the recommendations
Advocates are essential partners in engaging patients to help achieve the scientific priorities.
cancer.gov/brp Blue Ribbon Panel Report 2016
Policy issues identified by the BRP as barriers (e.g. coverage and reimbursement; uniform informed consent) have been forwarded to the Task Force for consideration.
Implementation will depend on the extent to which these barriers are addressed.
Extent and rate of implementation will depend on Congressional appropriations
Next Steps
Cancer data ecosystem
Well characterized research data
sets
Cancer cohorts Patient data
EHR, lab data, imaging, PROs, smart devices,
decision support
Learning from everycancer patient
Active researchparticipation
Research informationdonor
Clinical ResearchObservational studies
ProteogenomicsImaging dataClinical trials
Discovery Patient engaged Research
SurveillanceBig Data
Implementation research
SEER
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Cancer Research Data Commons Ecosystem
GenomicData Commons
Data StandardsValidation and Harmonization
ImagingData Commons
ProteomicsData Commons
Clinical Data Commons
(Cohorts / Indiv.)
SEER(Populations)
Data Contributors and Consumers
Researchers PatientsClinicians
Institutions
38
http://cancer.gov/brp
Questions?
www.cancer.gov/brp