Cancer Moonshot Blue Ribbon Panel Report.

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1 Cancer Moonshot Blue Ribbon Panel Report

2 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)

3 Vice President s Cancer Moonshot Workflow Vice President s Office Cancer Moonshot Federal Task Force NCI/NIH NCAB Blue Ribbon Panel Working Groups

4 Federal Task Force Goals Accelerate our understanding of cancer, its prevention, early detection, treatment and cure; Support greater access to new research, data, and computational capabilities; Improve patient access and care; Identify and address any unnecessary regulatory barriers and consider ways to expedite administrative reforms; Identify opportunities to develop public-private partnerships and increase coordination of the Federal Government s efforts with the private sector, as appropriate. (Presidential Memo 2016)

5 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, convene workshops, and conduct other activities. Findings and recommendations of the Panel will be reported to the NCAB. The NCAB will use the Panel s findings and recommendations to provide final recommendations to the NCI Director. (Presidential Memo 2016)

6 Blue Ribbon Panel 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 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 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 Edith Mitchell Thomas Jefferson Univ. *NCAB/BSA member

7 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. The NCAB will use the Panel s findings and recommendations to provide final recommendations to the NCI Director. (Presidential Memo 2016)

8 BRP Working Groups Working Group Co-Chair NCI Staff Cancer Immunology Precision Prevention and Early Detection Tumor Evolution Clinical Trials Implementation Sciences Pediatric Cancer Enhanced Data Sharing Liz Jaffee, Jim Allison Mary Beckerle, Jennifer Pietenpol Chi Dang, Levi Garraway Charles Sawyers, Mitch Berger Elena Martinez, Augusto Ochoa Peter Adamson Jim Downing Angel Pizarro Gaddy Getz Toby Hecht, Kevin Howcroft Elisa Woodhouse Tracy Lively Joanna Watson, Suresh Mohla, Tony Dickherber Jeff Hildesheim Meg Mooney Bob Croyle, Worta McCaskill- Stevens Judy Mietz Malcolm Smith Juli Klemm Betsy Hsu, Jennifer Couch

9 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 Over 1600 responses total One-on-one public input: BRP Listening sessions Professional conferences

10 Overview of Blue Ribbon Panel Report The Report summarizes the 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 will include all recommendations in their entirety at

11 Cross-Cutting Themes Health disparities research Prevention National network of patient biological and clinical data Biomarkers Development of technology and preclinical models Data sharing, analytics and predictive computational modeling Collaboration; public-private partnerships

12 Summary of the Recommendations A. Establish a network for direct patient involvement in cancer research: Cancer patients can join a new national network that will provide them with a genetic profile of their own cancer and let them preregister for clinical trials, so they can be contacted when a trial for which they may be eligible opens B. Cancer immunotherapy clinical trials network. Organize a network to discover and evaluate novel immune-based approaches for adult and pediatric cancers, and eventually develop new cancer prevention vaccines for children and adults 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 and how to prevent resistance

13 Summary of the Recommendations D. Creation of a national cancer data ecosystem. Create an ecosystem to collect, share, and interconnect datasets through links many of the nation s largest data repositories, which would enable onestop, free access for researchers, doctors, and diverse patient populations E. Intensify research on the major drivers of childhood 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.

14 Summary of the Recommendations (continued) 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. Increasing testing for hereditary cancer syndromes in people with certain types of cancer and their family members, so those identified as high risk can begin early prevention or screening efforts H. Retrospective analysis of biospecimens from patients treated with standard of care. Analysis of existing tumor tissue from patients, including those from racial and ethnic minority groups, who received standard of care treatment, stored at biobanks around the country, may enable discovery of genetic and other factors that distinguish which individuals would benefit from standard care versus experimental treatment in a clinical trial.

15 Summary of the Recommendations (continued) I. Creation of human tumor atlas. Catalog the evolution of genetic lesions and cellular interactions in tumor/immune/other cells in tumor microenvironment from the earliest detected lesions to metastasis to develop predictive models of tumor progression J. Development of new enabling technologies. Support development of technologies, through increased public-private sector collaboration, to accelerate testing of therapies and tumor characterization.

16 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 Translational Network A national pediatric immunotherapy translational network to discover and evaluate novel immunotherapies 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

17 Policy issues for further consideration Coverage and reimbursement Privacy and consent with regard to patient data Fragmentation of the delivery of patient care in the community The need to improve the clinical trials system Incentives to encourage pediatric drug development New federal research funding models Barriers to data sharing Ensuring that racial and ethnic minorities, as well as other underserved populations, are adequately represented when implementing policy-related recommendations

18 21 st Century Cures Bill Signed into law on December 13, 2016 $4.8B in new funding for the NIH $1.5B for Precision Medicine Initiative $1.8B over seven years for the Beau Biden Moonshot $500M in new funding for the FDA Modernization of clinical trials and how safety/efficacy data is accumulated/analyzed Supporting increased utilization of biomarkers More flexible approaches in reviewing medical devices for breakthrough technologies

19 NCI Funding Opportunity Announcements (FOAs) FOAs out on Therapeutic resistance, Immunotherapy New FOAs coming on Pediatric immunotherapy, Fusion oncoproteins, Technology development, Risk stratification, Tumor evolution, Cancer prevention Plan is to continue to release new FOAs over time period of funding

20 Vision for the future for cancer research and treatment Ethnically and racially diverse groups of patients contribute information about their cancer, obtain a genomic profile, learn what treatments might work best given their profile, and identify clinical trials that may be appropriate for them Doctors have access to information that better predicts treatment outcomes and helps control their patient s symptoms and side effects Researchers can identify possible targets for the development of new cancer treatments and preventive interventions, including immunotherapies, and learn more about how to avoid or counter cancer drug resistance in all patients

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