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, PhDwarren.kibbe@nih.gov

@wakibbe

NCI and The Cancer Moonshot

Town HallOctober 5, 2016

Douglas R. LowyActing Director, National Cancer Institute,

National Institutes of Health

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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

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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

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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

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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

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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)

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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

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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

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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

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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

Blue Ribbon Panel Report 2016

TEAG

www.cancer.gov/brp

Warren Kibbe, PhDwarren.kibbe@nih.gov

@wakibbe

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

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http://cancer.gov/brp

Questions?

www.cancer.gov/brp