Agenda and Speakers

University of Washington & Fred Hutchinson Cancer Center

Talk Title: Clinical Research in AML: Challenges and Opportunities

Dr. Percival is a hematologist with a focus on acute myeloid leukemia. She is an Associate Professor at the University of Washington and at the Fred Hutchinson Cancer Center in Seattle, WA. Dr. Percival earned her bachelor’s degree magna cum laude from Harvard University and her medical degree from Stanford University School of Medicine. She completed internship and residency at University of California, San Francisco, followed by fellowship in hematology/oncology at Stanford. She also received a master’s degree in epidemiology and clinical research from Stanford. She is the principal investigator on multiple clinical trials for AML. Dr. Percival is a member of two committees of the American Society of Hematology: the Committee on Practice and the Committee on Training. She is also an AML panel member for the National Comprehensive Cancer Network guidelines.

Dana Farber Cancer Institute, Harvard University

Talk Title: Clonal Hematopoiesis and Risk of Leukemia

Dr. Benjamin Ebert is the Chair of Medical Oncology at the Dana-Farber Cancer Institute, the George P. Canellos, MD and Jean S. Canellos Professor of Medicine at Harvard Medical School, a Howard Hughes Medical Institute Investigator, and an Institute Member of the Broad Institute. The Ebert laboratory focuses on the biology and therapy of hematologic malignancies. The laboratory has elucidated multiple novel mechanisms of targeted protein degradation, beginning with the mechanism of action lenalidomide, a derivative of thalidomide. Additional work has focused on the biology and genetics of myeloid malignancies, including the characterization of clonal hematopoiesis, a pre-malignant state for blood cancers. He is an elected member of the National Academy of Medicine, the American Society for Clinical Investigation and the Association of American Physicians. His awards include the Meyenburg Prize for Cancer Research, the Sjöberg Prize, and the Korsmeyer Award.

NHLBI, National Institutes of Health

Talk Title: Why Measure?

Dr. Hourigan is Chief of the Laboratory of Myeloid Malignancies at the NIH Intramural Program, where his translational laboratory focus is Measurable Residual Disease in AML. He also practices clinically on the acute leukemia inpatient service of Johns Hopkins Hospital. Dr. Hourigan graduated from Oxford University and completed clinical training at Guy’s and St Thomas’ Hospital in London and Johns Hopkins in Baltimore. In 2019 he received the Presidential Early Career Award for Scientists and Engineers.

Massachusetts General Hospital, Harvard University

Talk Title: Microenvironments and AML

David Scadden is the Gerald and Darlene Jordan Professor of Medicine at Harvard University. He is Professor and Chair emeritus of the Harvard University Department of Stem Cell and Regenerative Biology. He co-founded and co-directs the Harvard Stem Cell Institute with Prof. Douglas Melton. He is a hematologist/oncologist at the Massachusetts General Hospital where he founded and directs the Center for Regenerative Medicine and previously led the Hematologic Malignancies Program of the MGH Cancer Center. He is a member or fellow of the National Academy of Medicine, the American Academy of Arts and Sciences, the American Association for the Advancement of Science, the American College of Physicians, and a former member of the Board of External Experts for the National Heart, Lung and Blood Institute and the National Cancer Institute’s Board of Scientific Counselors. He is an Affiliate Member of the Broad Institute of Harvard and MIT and a Visiting Scholar of Pembroke College, University of Cambridge, England. He co-founded the public companies, Fate Therapeutics and Magenta Therapeutics and is a Director of Agios Pharmaceuticals and Editas Medicines. His work emphasizes using multidisciplinary approaches to define novel therapies for blood diseases. He is credited with having first experimentally defined a mammalian stem cell niche and its role in malignancy. His work on blood stem cells has led to new approaches to bone marrow transplantation now in clinical trial. He received the E. Donnall Thomas Award from the American Society of Hematology for his ‘pioneering work on the bone marrow microenvironment.’

University of Washington

Talk Title: Evolution of AML With Complex Karyotype

Doulatov lab seeks to understand how genetic alterations in normal hematopoietic stem cells (HSCs) drive the development of myeloid neoplasms, including myelodysplastic syndromes (MDS) and acute leukemias. We are interested in understanding the importance of order and sequence of premalignant somatic mutations and their impact on HSC differentiation and self-renewal. We utilize primary human cells and induced pluripotent stem cells (iPSCs) to create powerful models of hematological disorders (Doulatov et al. Sci Transl Med 2017). iPSCs are generated by reprogramming of MDS and AML patient samples, becoming a source of patient-specific HSCs. By combining reprogramming with genome sequencing and gene editing, we aim to reconstruct how pathogenic mutations cooperate to drive malignant transformation and develop new therapeutic interventions.

We are also interested in the biology of normal human hematopoiesis and erythropoiesis, with the focus on autophagy and mitophagy, key metabolic pathways for recycling damaged cellular components implicated in stem cell function and aging. Current goals involve understanding the role of autophagy in different hematopoietic cell types, defining cell-type specific molecular regulators of autophagy and mitophagy, and development of autophagy therapeutics.

Northwestern University

Talk Title: Germline Predisposition to Hematopoietic Malignancies

The Godley laboratory studies the molecular pathways that drive hematopoietic malignancies, with a focus on understanding how germline predisposition alleles contribute to individual and family risk as well as how covalently modified cytosines in DNA control cellular differentiation. Dr. Godley has contributed to the recognition of germline DDX41, ETV6, and CSF3R variants as risk factors to developing hematopoietic malignancies. She is also studying how deleterious germline RUNX1, CHEK2, and BRCA1/2 variants drive these cancers, especially considering how the development of clonal hematopoiesis and inflammatory pathways contribute to tumorigenesis. Dr. Godley has shown that germline contribution to hematopoietic malignancies occurs throughout the entire age range of life and is more common than previously anticipated, which has important implications for the allogeneic hematopoietic stem cell donor pool. Along with David Wu (UW), Dr. Godley co-chairs the Myeloid Malignancy Variant Curation Expert Panel, which has provided variant curation rules for RUNX1 that are now in use throughout the world and is currently developing similar rules for GATA2 and DDX41. Dr. Godley’s group also studies how hypoxia alters epigenetic modifications with hematopoietic stem and progenitor cells, especially during erythropoiesis.

Wayne State University

Talk Title: The Challenge of AML in “Olderly” People

Charles A. Schiffer, MD, is Emeritus Professor of Oncology and was previously the Joseph Dresner Chair for Hematologic Malignancies and the director of the Leukemia/Lymphoma Multidisciplinary Program at Wayne State University School of Medicine and the Karmanos Cancer Institute in Detroit, Michigan.

Dr. Schiffer earned his BA cum laude at Brandeis University and his M.D. at New York University School of Medicine. He completed his internship, residency, and chief residency in Internal Medicine at Bellevue Hospital under the auspices of New York University School of Medicine and had subsequent training and positions at the Baltimore Cancer Research Institute, National Cancer Institute and the University of Maryland School of Medicine, where he served as Chief of the Division of Hematology. He has also served as Chief of the Division of Hematology/Oncology and Director of Clinical Research at the Karmanos Cancer Institute.

Dr. Schiffer has authored and co-authored more than 350 articles and 80 book chapters on topics concerning the treatment of leukemia in adults, platelet transfusion, and granulocyte transfusion therapy, among others. He has served on the Editorial Boards for Blood, the Journal of Clinical Oncology, International Journal of Hematology, Transfusion Medicine Reviews and Transfusion, and reviews articles for multiple journals. Committee memberships have included Chairman of the Leukemia Committee of the Cancer and Leukemia Group B, Chairman of the Food and Drug Administration Oncologic Drug Advisory Committee, member of the American Board of Internal Medicine – Medical Oncology Board, and grant reviews for the NCI, ASH, DOD, ASCO and Leukemia/Lymphoma Society of America. Dr. Schiffer has been named among Castle Connelly’s “Best Doctors in America,” and Newsweek’s “Best Cancer Specialists in the US.” In 2006, he received the Dr. John J. Kenney Award from the Leukemia/Lymphoma Society of America and the Celgene Award for Career Achievement in Hematology. He has received numerous teaching awards from the School of Medicine and was recently inducted into the Academy of Scholars of Wayne State University, the highest recognition accorded to academic faculty at the University.

Georgia Cancer Center

Talk Title: IDH1 Inhibitors for AML: Is Olutasidenib a Valuable Addition?

Dr. Jorge Cortes’ career has been focused in clinical research with particular focus on new drug development for myeloid malignancies. He has been involved in the development in tyrosine kinase inhibitors for CML, leading the approval of bosutinib and ponatinib as well as omacetaxine. He has also led trials that resulted in the approval of new agents for acute myeloid leukemia including glasdegib and olutasidenib; quizartinib will also likely soon be approved. Recent work has expanded to assess and address health care disparities in patient care and clinical research in hematologic malignancies and other cancers. This work has focused on the inequality in enrollment of patients in clinical trials based in race, sex and place of residence, both in the US and globally.

Washington University School of Medicine

Talk Title: Current State of Immunotherapy for AML and Future Directions

John F. DiPersio, MD, PhD: Work in the DiPersio laboratory focuses on fundamental and translational aspects of leukemia, lymphoma and stem cell biology. These studies include identification of genetic abnormalities in human leukemias, understanding processes involving stem cell and leukemia cell trafficking and clinical and translational programs in both leukemia/MDS, lymphoma/myeloma and stem cell transplantation. As Deputy Director of the Siteman Cancer Center, I oversee all clinical and basic science research in the cancer center, serve as a mentor of trainees and junior faculty. My personal research has focused on the role of stem cell transplantation and novel targeted interventions to alter the natural history of AML and other hematological malignancies. These studies have utilized bench-to-bedside mechanistic and preclinical modeling studies followed by early phase clinical trials. They have focused on targeting key elements of the hematopoietic niche for optimal stem cell mobilization and chemosensitization, mitigating GvHD in T replete transplants, understanding the genomic alterations in de novo and relapsed AML and developing immunotherapeutics, CART and gene edited CART for the treatment of AML, T-ALL and T and B/T-NHL.