CCAF Overview April 13, 2014 Ze’ev Ronai, Ph.D . Scientific Director, La Jolla Deputy Director, NCI Cancer Center

1. Cancer Center @ Sanford-Burnham Medical Research Institute CCAF Overview April 13, 2014 Ze’ev Ronai, Ph.D. Scientific Director, La Jolla Deputy Director, NCI Cancer Center

2. 10-year Strategic Vision 2014-2023 Our MISSION Discover the cause of disease Invent cures 47 of 64 SBMRI faculty are members of the cancer center engaged in cancer research

3. 10-year Strategic Vision 2014-2023 Our COMPETITIVE ADVANTAGE Combined strength Discovery Research Therapeutics Discovery

4. 10-year Strategic Vision 2014-2023 Our CORE VALUE Collaboration Collaboration extended to include members of the C3

5. 10-year Strategic Vision 2014-2023 Our VISION Pillar 1 Pillar 2 Discovery Research Therapeutics Discovery • Generation of new knowledge of biology and disease mechanisms • Increase human-disease relevance of our discovery research • Speed the movement of laboratory discoveries into therapeutics discovery • Small-molecule drug discovery platform will be the engine for accelerating therapeutics discovery Therapeutic Discovery is available for members of the C3

6. Snapshot of the SBMRI Cancer Center 1976 La Jolla Cancer Research Foundation founded by William & Lillian Waterman Fishman in La Jolla, CA 1981 becomes an NCI-designated Research Cancer Center. One of seven NCI basic Research Cancer Centers NCI-Cancer Center faculty are 73% (47/64) of SBMRI faculty 61 post docs, 20 grad students Largest-but-one NCI basic science cancer center (faculty and budget) with total annual NCI funding >$20M 134 cancer relevant publications per year

7. SBMRI Cancer Center Mission • The SBMRI Cancer Center will be a national leader in the battle to overcome cancer as a cause of human suffering and death. • conducting paradigm shifting, multidisciplinary basic research into the causes, prevention and treatment of cancer • rapidly translating our discoveries into the clinic for the benefit of cancer patients everywhere • educating and training the next generation of cancer scientists

8. Characteristics - SBMRI Cancer Center Outstanding high impact science leading to novel therapies Largest academic infrastructure for cancer drug discovery Addressing timely unmet medical needs Located in the second largest biotech cluster in the US

9. Engaging minority and diverse students in cancer research SBMRI CC hosts six Fresno State summer students; who work in three CC laboratories under the supervision of CC faculty. The objective is to recruit promising minority students to doctoral programs in cancer research, preferably at Sanford-Burnham. Fresno State Program – P20 SBMRI CC hosts 10 Preuss School rising seniors as summer interns for 6 weeks each summer supervised by CC faculty The objective is to provide mentoring, laboratory skills, and experience in an environment that inspires students to seek an undergraduate major in the biological sciences. Preuss High School Program – P30 supplement

10. SBMRI Cancer Community Advisory Board Mission To promote a dynamic exchange of information between the Sanford-Burnham scientific community, patients and their supporters. • Presentations at Cancer Center Faculty Retreats • Provide advocacy support for research proposals, as well as involvement in project development. • Host National Cancer Survivors Day event (6/2014) • Host on-campus open houses to promote CC research and provide patients, their supporters and the public better insight into the importance of our science

11. STRIVE Translational Initiative Goal: To provide advice and support to move SBMRI innovative drug or diagnostic projects to the next level Bi-monthly meetings attended by faculty, students and representatives from La Jolla’s biotech and pharma community. Projects Inhibitors of fatty acid synthase as cancer agents* - Dr. Jeff Smith NEDD8 activating enzyme inhibitor for cancer therapy* - Dr. Matt Petroski Targephrin: Drug delivery selectively targeting cancer cells * - Dr. Maurizio Pellecchia Cyclic EphA4 peptide antagonists for cancer stem cells - Dr. Elena Pasquale *Funded projects (milestone driven) ~ $50 K.

12. NCI Basic Cancer Centers Total NCI funding 2013 in $M r CC Faculty 7 87 30 31 30 48 27 Source: NIH Online Research Portfolio 2013

13. Data: Membership and Themes (47 + 8 adjunct) Program 1 Program 2 Program 3 Tumor Initiation and Maintenance Cell Death and Survival Networks Tumor Microenvironment and Metastasis (TIM) (CDSN) (TMEM) Robert Wechsler- - Jorge Moscat, PhD Sara Courtneidge, PhD Reya, PhD Program Leader Themes Program Leader Themes Program Leader Themes Metabolic and Stress Signaling Stem Cells and Development Modulation of Angiogenesis and Immune/Inflammatory 7 members 7 members 8 members Cell Growth Signaling Autophagy and Cell Death Cell Migration, Invasion and Metastasis 3 members 6 members 6 members New Intervention Points for Cancer Therapy Molecular Therapeutics RNA Biology 3 members 6 members 3 members

14. Sanford-Burnham Technology Infrastructure Complementing Expertise for the C3 members Animal Resources Animal Care & Procedures In Vivo Imaging & Analysis Tumor Analysis Cardiometabolic Phenotyping Model Organisms Cell Imaging & Analysis Cell Imaging Histopathology Flow Cytometry Structural Biology Crystallography Nuclear Magnetic Resonance Protein Production & Analysis Stem Cells Embryonic Stem Cells iPSCs & Disease Models Conrad Prebys Center For Chemical Genomics Assay Development High Throughput Screening High Content Screening Ultra-HTS Cell-Based Disease Modeling Cheminformatics Medicinal Chemistry Exploratory Pharmacology Genomics DNA Analysis Microarray & QPCR HT DNA sequencing Shared Resources Functional Genomics RNAi and cDNA screening Viral Vectors Proteomics & Metabolomics Proteomics Metabolomics Cancer Metabolism Bioinformatics & Data Management Bioinformatics Statistics 3D Computational Modeling

15. Cancer Centers Council 3C • http://sdcancercouncil.org SBMRI, UCSD Moores, Salk Institute Formally launched June 2013 • Shared Resources at internal rate +16% • Biorepository and Tissue Technology (Moores CC) • Viral Vectors (Salk Institute) • High Throughput Screening (SBMRI) • Pedal the Cause to support collaborative translational grants between the three institutions ($0.5M in its first year) • Annual joint scientific retreat • highlighting collaborative projects

16. Cancer Centers Council 3C • http://sdcancercouncil.org C3 Characteristics & goals: facilitate collaborative cancer research activities between the institutions; leveraging resources, with select shared resources available to all investigators at internal member rates organizing joint public and scientific initiatives facilitating interactions with other research entities in the public and private sector in and around San Diego. 9 inter-institutional projects are currently underway; 2 with The Salk Institute CC and 7 with UCSD. Extending and enhancing collaborations: >45 joint publications

17. SBMRI Cancer Center Access to human tumor tissues* • Agreements with: • Florida Hospital System, Orlando 1,2,3 • pancreas, colon, prostate, breast • University of Colorado, Denver2,3 • melanoma • Vancouver Prostate Centre2 • prostate • Moores Cancer Center C3, UCSD1,3,4 • glioblastoma, medulloblastoma, pancreas, colon, • breast, liver • MD Anderson Cancer Center3 • colon • Yale University 1,3 • lung, melanoma • Molecular Research (La Jolla)4 • colon, prostate, breast, lung • Scripps Health System (San Diego) • lung, colon 1,2,3 • 1fresh tissue, 2TMA, 3PDX mice, 4frozen tissue digest • *Coordinated by the Tissue and Histopathology Shared Resource

18. C3

19. CCAF Discussion