Cambridge Healthtech Institute’s Third Annual
Targeting Cancer Stem Cells
New Opportunities for Oncology Therapeutics
Part of the 21st Annual Molecular Medicine Tri-Conference

February 13-14, 2014 | Westin St. Francis | San Francisco, CA

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Friday, February 14

8:00 am Morning Coffee


8:25 Chairperson’s Remarks

8:30 Strategies to Develop Agents that Preferentially Target Cancer Stem Cells: Clinical Candidates Targeting FAK and PI3K/mTOR

Jonathan A. Pachter, Ph.D., Vice President & Head of Research, Verastem, Inc.

9:00 Antibody-Based Approaches to Targeting Proliferating and Quiescent Cancer Stem Cells

Robert J. Tressler, Ph.D., Vice President, Research & Development, Cellerant Therapeutics, Inc.

9:30 Challenges in Designing Clinical Trials with Stem Cell-Directed Therapies

Anne F. Schott, M.D., Associate Professor, Department of Internal Medicine, University of Michigan

Laboratory research has led to the identification of clinical compounds that target the cancer stem cell population. Clinical trials are being developed to evaluate these stem cell directed therapies, often in combination with a standard treatment such as chemotherapy. The combination therapy paradigm exposes the challenge of demonstrating efficacy and specificity of stem cell targeted agents. New clinical/translational trial endpoints and assays will be necessary to advance the field.

10:00 FEATURED ORAL POSTER PRESENTATION: How to Kill Cancer Stem Cells Without Killing Normal Stem Cells in Adult Patients

William G. Thilly, Sc.D., Professor of Genetics, Toxicology and Biological Engineering, Department of Biological Engineering, Massachusetts Institute of Technology

Through sponsored research program at MIT a series of drugs were identified that specifically kill cancer stem cells but not non-stem cells in cell cultures derived from human pancreatic and colorectal tumors. Several of these drugs are slated for clinical experiments in which pancreatic cancer patients will be treated before surgery to remove their tumors. Using proprietary means the tumors will be dissected and the extent of cancer stem cell killing determined. Those drugs found to be lethal to pancreatic stem cells in patients will be used together in a proprietary regimen one facet of which is designed to overcome the expected resistance of mutant cancer stem cells to individual drugs.

10:30 Coffee Break with Exhibit and Poster Viewing

11:00 Cancer Stem Cell Strategies for Patient Specific Cancer Immunotherapy: Stopping Cancer in It’s Tracks

Andrew Cornforth, Ph.D., Cancer Stem Cell Program Manager, California Stem Cell, Inc.

An ideal source of antigens to stimulate an immune response in an immunotherapeutic approach is to use the patient’s own tumor. Limitations to this approach are the acquisition of tumor samples in adequate quantities and the lack of antigen sources from the most aggressive phenotypes, namely tumor stem cells. Our approach capitalizes on the recent developments in specific media formulations to isolate and propagate putative cancer stem cells from patient tumor samples to quantities necessary for loading dendritic cells.


11:30 Clinical and Preclinical Studies of Stemline Therapeutics’ Clinically Active Agents, SL-401 and SL-701, Directed at Cancer Stem Cells and Tumor Bulk of Hematologic and Brain Cancers

Eric K. Rowinsky, M.D., Head, Research & Development and CMO, Stemline Therapeutics, Inc.

Among its portfolio candidates, Stemline Therapeutics, Inc. is developing two clinical-stage candidates. SL-401 is a targeted therapy directed to the interleukin-3 receptor, which is overexpressed on the CSCs and tumor bulk of many hematologic cancers, whereas SL-701 is a cancer vaccine comprised of synthetic peptides, designed for immune system targeting of both CSCs and TB of brain cancers. These agents have produced regressions in advanced hematologic and brain malignancies, including complete responses and strong survival signals, in phase I/2. Pivotal trials with SL-401 will be initiated in AML and blastic plasmacytoid dendritic cell malignancy, a rare aggressive cancer and unmet medical need, whereas phase 2 trials evaluate SL-701 in children and adults with high-grade glioma.

12:00 pm Dendritic Cell Vaccines Targeting Cancer Stem Cells

John S. Yu, M.D., Professor & Vice Chairman, Neurosurgery, Cedars-Sinai Medical Center; Chairman & CSO, ImmunoCellular

We have developed dendritic cell vaccines for brain cancer. Lead candidate ICT-107 is currently being evaluated in a Phase II, randomized clinical trial. We will present an update on this trial, as well as clinical and immunologic data on the CD133 targeting DC vaccine (ICT-121). A DC vaccine for ovarian cancer has undergone FDA acceptance of an IND and development of a multi-institutional Phase I/II trial will be discussed.

12:30 Close of Symposium

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