Invited Session 4: Strategies to develop treatments for rare tumors
Chair: Shivaani Kummar, Oregon Health & Science University
Dr. Kummar is DeArmond Endowed Chair of Cancer Research, Division Chief of Hematology and Medical Oncology, co-Director of the Center of Experimental Therapeutics, and Deputy Director for Clinical and Translational Research, Knight Cancer Institute, Oregon Health & Science University. In her prior positions she served as head of Early Clinical Trials Development in the Office of the Director, Division of Cancer Treatment and Diagnosis, National Cancer Institute, and Professor and Director of the Phase I Clinical Research and Translational Oncology Programs, Stanford University. She specializes in conducting pharmacokinetic and pharmacodynamic driven first-in-human trials tailored to make early, informed decisions regarding the suitability of novel molecular agents for further clinical investigation. She is a member of scientific planning committees of national and international professional organizations and has authored over 150 peer reviewed publications, 9 book chapters, and co-edited a book on ‘Novel Designs of Early Phase Trials for Cancer Therapies’. She is currently a member of the American Association for Cancer Research Exploratory IND/Phase 0 Clinical Trials Task Force, Cancer Progress Report 2022 Steering Committee, and co-Chair of the Clinical Trials Committee.
Developing treatments for rare tumors: Challenges and Opportunities
Speaker: Shivaani Kummar, Oregon Health & Science University
Dr. Kummar is DeArmond Endowed Chair of Cancer Research, Division Chief of Hematology and Medical Oncology, co-Director of the Center of Experimental Therapeutics, and Deputy Director for Clinical and Translational Research, Knight Cancer Institute, Oregon Health & Science University. In her prior positions she served as head of Early Clinical Trials Development in the Office of the Director, Division of Cancer Treatment and Diagnosis, National Cancer Institute, and Professor and Director of the Phase I Clinical Research and Translational Oncology Programs, Stanford University. She specializes in conducting pharmacokinetic and pharmacodynamic driven first-in-human trials tailored to make early, informed decisions regarding the suitability of novel molecular agents for further clinical investigation. She is a member of scientific planning committees of national and international professional organizations and has authored over 150 peer reviewed publications, 9 book chapters, and co-edited a book on ‘Novel Designs of Early Phase Trials for Cancer Therapies’. She is currently a member of the American Association for Cancer Research Exploratory IND/Phase 0 Clinical Trials Task Force, Cancer Progress Report 2022 Steering Committee, and co-Chair of the Clinical Trials Committee.
Designing and conducting trials for adults with rare CNS tumors- challenges, opportunities, lessons learned
Speaker: Marta Penas-Prado, NIH/NCI
Dr. Penas-Prado is a neuro-oncologist focused on caring for adults with rare brain and spine tumors such as ependymoma, medulloblastoma, and others in the NCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors) program at the Neuro-Oncology Branch (NOB), Center for Cancer Research (CCR), National Cancer Institute (NCI), National Institutes of Health (NIH). Her clinical research seeks to uncover the molecular underpinnings of rare central nervous system (CNS) tumors, better understand these diseases, and develop clinical trials to develop more effective therapies. Rare tumors frequently present clinical challenges due to issues such as poorly defined options for upfront treatment and recurrence and limited access to specialized care and clinical trials.
Beyond the NOB, Dr. Penas-Prado enjoys collaborating with a multidisciplinary team of neuropathologists, neuroradiologists, neurosurgeons, radiation oncologists, genetic counselors, and others to provide the best possible care for her patients and establish a care team with their local physicians. She also leads a virtual multidisciplinary, multi-institutional tumor board to discuss complex neuro-oncology cases.
Understanding the natural history and accelerating better therapies for children, adolescents and young adults with rare tumors
Speaker: Brigitte Widemann
Practical Considerations in Selecting Patient Reported Outcomes and Alternative Outcomes Assessments in Rare Disease – A Sponsor’s Perspective
Speaker: Timothy Bell, SpringWorks Therapeutics, Inc.
Tim Bell is Executive Director of the Medical Research and Value Demonstration Team at SpringWorks Therapeutics, Inc., a commercial-stage biopharmaceutical company that applies a precision medicine approach to developing and delivering life-changing medicines. He has over 25 years of experience in Health Economics, Outcomes Research, Market Access, and Medical Affairs across industry, consulting, and academia. He has authored or co-authored multiple peer-reviewed articles as well as abstracts/posters/presentations across a broad range of therapeutic areas (e.g., rare disease and oncology) and in a variety of disciplines (e.g., health economics and outcomes research, real world data, epidemiology, patient reported outcomes and quality of life, prospective and retrospective non-interventional studies and registries, comparative effectiveness research, and clinical trials). He also has a diverse range of geographic experience, serving in global roles as well as dedicated regional roles covering the US, Europe, Latin America, Canada, Japan, Australia, and South Korea.