CCAF Overview

April 13, 2014

Ze’ev Ronai, Ph.D.Scientific Director, La Jolla

Deputy Director, NCI Cancer Center

Cancer Center @ Sanford-Burnham Medical Research Institute

Our MISSIONInvent curesDiscover the cause of disease

10-year Strategic Vision 2014-2023

47 of 64 SBMRI faculty are members of the cancer center engaged in cancer research

Combined strength

Therapeutics DiscoveryDiscovery Research

Our COMPETITIVE ADVANTAGE

10-year Strategic Vision 2014-2023

Collaboration

Our CORE VALUE

10-year Strategic Vision 2014-2023

Collaboration extended to include members of the C3

Our VISIONPillar 1 Pillar 2

• Speed the movement of laboratory discoveries into therapeutics discovery

• Small-molecule drug discovery platform will be the engine for accelerating therapeutics discovery

Therapeutics DiscoveryDiscovery Research

• Generation of new knowledge of biology and disease mechanisms

• Increase human-disease relevance of our discovery research

10-year Strategic Vision 2014-2023

Therapeutic Discovery is available for members of the C3

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

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

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

Engaging minority and diverse students in cancer research

Fresno State Program – P20

Preuss High School Program – P30 supplement

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.

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.

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

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

1. Inhibitors of fatty acid synthase as cancer agents* - Dr. Jeff Smith

2. NEDD8 activating enzyme inhibitor for cancer therapy* - Dr. Matt Petroski

3. Targephrin: Drug delivery selectively targeting cancer cells * - Dr. Maurizio Pellecchia

4. Cyclic EphA4 peptide antagonists for cancer stem cells - Dr. Elena Pasquale

*Funded projects (milestone driven) ~ $50 K.

NCI Basic Cancer Centers

Jack-son Lab

Purdue Salk Cold Spring Harbo

Wistar SBMRI MIT0

5

10

15

20

25

Total NCI funding 2013 in $M

CC Faculty 7 87 30 31 30 48 27

Source: NIH Online Research Portfolio 2013

r

Data: Membership and Themes (47 + 8 adjunct)

Program 1 Tumor Initiation and Maintenance

(TIM)

Program 2 Cell Death and Survival Networks

(CDSN)

Program 3 Tumor Microenvironment and Metastasis

(TMEM)

Robert Wechsler--

Reya, PhD

Program Leader

Themes

Stem Cells and Development

Cell Growth Signaling

RNA Biology

Jorge Moscat, PhD

Metabolic and Stress Signaling

Autophagy and Cell Death

Molecular Therapeutics

Sara Courtneidge, PhD

Modulation of Angiogenesis and Immune/Inflammatory

Cell Migration, Invasion and Metastasis

New Intervention Points for

Cancer Therapy

7 members

6 members

3 members

7 members

3 members

8 members

6 members

3 members

Program Leader

Themes

Program Leader

Themes

6 members

Sanford-Burnham Technology InfrastructureComplementing Expertise for the C3 members

Animal ResourcesAnimal Care & ProceduresIn Vivo Imaging & AnalysisTumor AnalysisCardiometabolic PhenotypingModel Organisms

Cell Imaging & AnalysisCell ImagingHistopathology Flow Cytometry

GenomicsDNA AnalysisMicroarray & QPCRHT DNA sequencing

Proteomics & MetabolomicsProteomicsMetabolomicsCancer Metabolism

Structural BiologyCrystallographyNuclear Magnetic ResonanceProtein Production & Analysis

Bioinformatics &Data ManagementBioinformaticsStatistics3D Computational Modeling

Conrad Prebys CenterFor Chemical GenomicsAssay DevelopmentHigh Throughput ScreeningHigh Content ScreeningUltra-HTSCell-Based Disease ModelingCheminformaticsMedicinal ChemistryExploratory Pharmacology

Functional GenomicsRNAi and cDNA screeningViral Vectors

Stem Cells Embryonic Stem Cells iPSCs & Disease Models

SharedResources

Cancer Centers Council 3C

Shared Resources at internal rate +16% 1. Biorepository and Tissue Technology (Moores CC)2. Viral Vectors (Salk Institute)3. 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

SBMRI, UCSD Moores, Salk Institute Formally launched June 2013

http://sdcancercouncil.org

Cancer Centers Council 3C

C3 Characteristics & goals:

1. facilitate collaborative cancer research activities between the institutions;

2. leveraging resources, with select shared resources available to all investigators at internal member rates

3. organizing joint public and scientific initiatives

4. 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

http://sdcancercouncil.org

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

C3

CCAF Discussion