Rare Cancers Prevention and Interception Workshop (Agenda)

Welcome

Altaf Mohammed, Ph.D., Workshop Co-Organizer, DCP, NCI

Introductory Remarks

Kimryn Rathmell, M.D., Ph.D., Director, NCI

Phil Castle, Ph.D., Director, Division of Cancer Prevention, NCI

Plenary Talk

Brigitte C. Widemann, M.D., Center for Cancer Research (CCR), NCI

Overview of Rare Cancers and Highlights of NCI Efforts

Session I

Overview of Rare Cancers: Current Status

Co-Chairs: Taylor Sundby, M.D., and Robert Shoemaker, Ph.D.

Sharon Savage, M.D., Division of Cancer Epidemiology and Genetics, NCI

The NCI Cancer Prevention and Interception Clinic: Conception and Vision

Uri Tabori, M.D., The Hospital for Sick Children, University of Toronto

Clinical and biological updates on replication repair deficiency syndromes

Payal Khincha, M.B.B.S., M.S.H.S., Division of Cancer Epidemiology and Genetics, NCI

Multidisciplinary approaches to germline cancer predisposition: the Li-Fraumeni syndrome perspective

Paul P. Liu, M.D., Ph.D., National Human Genome Research Institute

Inherited predisposition to hematologic malignancies - NIH natural history study of RUNX1-FPDMM

Samra Turajlic, M.D., Ph.D., The Francis Crick Institute

Replaying the tape of malignant transformation in VHL disease

Session II

Emerging Opportunities for Rare Cancers Prevention and Interception

Co-Chairs: Altaf Mohammed, Ph.D., and Payal Khincha, M.B.B.S., M.S.H.S.

Sarah Blagden, M.D., Ph.D., University of Oxford, London

Metformin pharmacoprevention trial in Li-Fraumeni syndrome (MILI)

Yurong Song, Ph.D., Frederick National Laboratory for Cancer Research

Preclinical studies for Lynch syndrome cancer prevention and interception

Irene Ghobrial, M.D., Dana Farber Cancer Institute

Early Detection and Interception in Myeloma

David Largaespada, Ph.D., University of Minnesota

Proteomic and immunopeptidomic characterization of the MPNST cell surface for targeted therapy discovery

Adjourn

Kajal Biswas, Ph.D., Workshop Co-Organizer, DCP, NCI

Welcome

Sagar Ghosh, Ph.D., MBA., Workshop Co-Organizer, CDMRP, DoD

Introductory Remarks

Colonel Sarah B. Goldman, Ph.D., Director, CDMRP, DoD

Session III

Networking - scientists, advocacy groups, and healthcare providers

Moderators: Sagar Ghosh, Ph.D., MBA., and Payal Khincha, M.B.B.S., M.S.H.S.

Patient Advocate Corrie Painter, Ph.D., Broad Institute

Driving discoveries in rare cancers through social connections

Patient Advocate Isis Sroka, Ph.D. Franconi Anemia Research Fund

Empowering Fanconi Anemia Advocates to Drive Innovation in Rare Cancer Research

Patient Advocate Katrin Ericson, Ph.D., RUNX1 Research Program

Synergizing Science and Advocacy: Strategies to Optimize Collaboration and Improve Patient Engagement

Additional Discussion to Follow in Session VI

Session IV

Rare Cancers Clinical Trials Updates

Co-Chairs: Martha Donoghue, M.D., and Anju Singh, B.V.Sc, Ph.D.

Theodore W. Laetsch, M.D., Children's Hospital of Philadelphia

Rare Tumor Clinical Trials in the Children's Oncology Group

Alice Chen, M.D., Division of Cancer Treatment and Diagnosis, NCI

Rare tumor trials in a phase I clinic: Ideal Match

Lunch

Eduardo Vilar-Sanchez, M.D., Ph.D., MD Anderson Cancer Center

The Current Landscape of Cancer Interception Clinical Trials in Lynch Syndrome

Andrea M. Gross, M.D., CCR, NCI

Challenges of Clinical Trial Design for Prevention in Rare Diseases

Martha Donoghue, M.D., The Food and Drug Administration

Regulatory Considerations for Clinical Trials in Rare Cancers

Session V

Challenges and Opportunities in Rare Cancers Research: Biology, Targets and Prevention

Co-Chairs: Kajal Biswas, Ph.D., and Grace Ault, Ph.D.

Kevin B. Jones, M.D., University of Utah

Interrogating epigenomic mechanisms in rare cancers with mouse genetic models

William Foulkes, Ph.D.; M.B.B.S., McGill University

Rare and unexplained familial tumors: past, present, future

Break

Steven Rhodes, M.D., Ph.D., Indiana University

Decoding NF1 Peripheral Nerve Sheath Tumor Heterogeneity: Insights from Genetically Engineered Mice

Thomas D. Wang, M.D. Ph.D., University of Michigan

Early Detection of Barrett's Neoplasia using Multi-Valent Interactions

Robert Shoemaker, Ph.D., Division of Cancer Prevention, NCI

Feasibility of Using Frameshift Mutations in Peripheral Blood as a Biomarker for Surveillance of Lynch Syndrome

Session VI

Brainstorming and Future Directions

Moderators: Anirban Das, M.B.B.S., M.D., D.M., and Altaf Mohammed, Ph.D.

Panelists: Brigitte Widemann, Robert Shoemaker, Sharon Savage, Martha Donoghue, Anirban Das, Payal Khincha, David Largaespada, William Foulkes, Theodore Laetsch, Katrina Ericson, Alice Chen, Paul Liu, Andrea Gross

Closing Remarks and Adjourn

Kajal Biswas / Altaf Mohammed / Sagar Ghosh