Presentation to the Fall CI4CC meeting in Utah. CI4CC Moonshot Blue Ribbon Panel Report. Highlights of Vice President Biden's Cancer Moonshot and the NCI Blue Ribbon Panel Recommendations.

1. Blue Ribbon Panel Report 2016
CI4CC in Utah
October 10th, 2016
www.cancer.gov/brp
Warren A. Kibbe, PhD
warren.kibbe@nih.gov
@wakibbe

2. NCI and The Cancer Moonshot
Town Hall
October 5, 2016
Douglas R. Lowy
Acting Director, National Cancer Institute,
National Institutes of Health

3. 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. 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

6. Changes in RPG pool: FY12-FY16 and beyond

7. 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

8. ■ White
♦ Black
▲ API
* AI/AN
● Hispanic*
*Hispanic is not mutually exclusive from other groups
Incidence data from SEER 13 1992-2010, Mortality data from NCHS
8
*Hispanic is not mutually exclusive from other groups
Incidence data from SEER 13 1992–2013, Mortality data from NCHS
Colon & Rectum
0
10
20
30
40
50
60
70
80
Incidencerateper100,000
Year of diagnosis
SEER Incidence
0
10
20
30
40
50
60
70
80
Mortalityrateper100,000
Year of death
US Mortality

9. 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

10. ■ White
♦ Black
▲ API
* AI/AN
● Hispanic*
*Hispanic is not mutually exclusive from other groups
Incidence data from SEER 13 1992-2010, Mortality data from NCHS
10
*Hispanic is not mutually exclusive from other groups
Incidence data from SEER 13 1992–2013, Mortality data from NCHS
Myeloma
0
3
6
9
12
15
Incidencerateper100,000
Year of diagnosis
SEER Incidence
0
3
6
9
12
15
Mortalityrateper100,000
Year of death
US Mortality

11. 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

12. 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. 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. 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. 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. 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. 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

18. Blue Ribbon Panel Report 2016
TEAG
www.cancer.gov/brp
Warren Kibbe, PhD
warren.kibbe@nih.gov
@wakibbe

19. 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.

20. Co-Chairs
Tyler Jacks* Elizabeth Jaffee* Dinah Singer
MIT Johns Hopkins NCI
Peter C. Adamson, M.D.*
Children's Hospital of Philadelphia
James Allison
MD Anderson
David Arons
National Brain Tumor Society
Mary Beckerle
Univ. of Utah
Mitchel Berger*
UCSF
Jeffrey Bluestone
Parker Institute
Chi Dang*
U. Penn
Mikael Dolsten
Pfizer
*NCAB/BSA member
Augusto Ochoa
Louisiana State Univ.
Jennifer Pietenpol
Vanderbilt Univ.
Angel Pizzaro
Amazon Web Services
Barbara Rimer
UNC
Charles Sawyers*
MSK
Ellen Sigal
Friends of Cancer Research
Patrick Soon-Shiong
NantWorks
Wai-Kwan Alfred Yung
MD Anderson
James Downing
St. Jude Hospital
Levi Garraway
Harvard Medical School
Gad Getz
Broad Institute
Laurie Glimcher
Weill Cornell
Lifang Hou
Northwestern
Neal Kassell
Univ. Va.
Elena Martinez*
UCSD
Deborah Mayer
UNC
Edith Mitchell
Thomas Jefferson Univ.
Blue Ribbon Panel

21. 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)

22. 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 Woodhouse
Tracy Lively
Tumor Evolution Chi Dang,
Levi Garraway
Joanna Watson, Suresh Mohla,
Tony Dickherber
Clinical Trials Charles Sawyers,
Mitch Berger
Jeff Hildesheim
Meg Mooney
Implementation Sciences Elena Martinez,
Augusto Ochoa
Bob Croyle, Worta McCaskill-
Stevens
Pediatric Cancer Peter Adamson
Jim Downing
Judy Mietz
Malcolm Smith
Enhanced Data Sharing Angel Pizarro
Gaddy Getz
Juli Klemm
Betsy Hsu, Jennifer Couch
BRP Working Groups

23. 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

24. cancer.gov/brp Blue Ribbon Panel Report 2016
Scientific and Community Outreach Activities
Goal:
 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!

25. 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)

26. 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

27. 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

28. 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.)

29. cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the Recommendations
A. 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.

30. cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the Recommendations
E. 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.

31. 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.

32. cancer.gov/brp Blue Ribbon Panel Report 2016
Summary of the Demonstration Projects
Prevention: 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

33. 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

34. 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.

35. 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

36. Cancer data ecosystem
Well characterized
research data sets Cancer cohorts Patient data
EHR, lab data, imaging,
PROs, smart devices,
decision support
Learning from every
cancer patient
Active research
participation
Research information
donor
Clinical Research
Observational studies
Proteogenomics
Imaging data
Clinical trials
Discovery
Patient engaged
Research
Surveillance
Big Data
Implementation research
SEER

37. 37
Cancer Research Data Commons Ecosystem
Genomic
Data Commons
Data Standards
Validation and Harmonization
Imaging
Data Commons
Proteomics
Data Commons
Clinical Data
Commons
(Cohorts / Indiv.)
SEER
(Populations)
Data Contributors and Consumers
Researchers PatientsClinician
s
Institutions

38. 38
http://cancer.gov/brp

39. Questions?
www.cancer.gov/brp