Marquee Sessions and Lectures

Announcement of Awards: Wallace H. Coulter Award for Lifetime Achievement in Hematology, ASH Mentor Awards, and ASH Award for Leadership in Promoting Diversity

WALLACE H. COULTER AWARD FOR LIFETIME ACHIEVEMENT IN HEMATOLOGY
ASH will recognize Rainer Storb, MD, of the Fred Hutchinson Cancer Center, with the Wallace H. Coulter Award for Lifetime Achievement in Hematology, the Society's highest honor. For more than 60 years, he has made groundbreaking achievements in hematopoietic stem cell transplantation (HSCT), a hallmark treatment for multiple blood disorders. He was instrumental in the creation and refinement of HSCT, as well as in advancing the understanding and treatment of acute and chronic graft-versus-host disease. His research has helped establish more effective and less toxic transplant approaches.

Dr. Storb’s interest in bone marrow led him to the lab of past ASH President and Nobel Laureate E. Donnall Thomas, MD, at the University of Washington, where he contributed to the development of HSCT and its subsequent translation to humans. They led the development of HSCT as a cure for aplastic anemia and other blood disorders, and, with Dr. Paul Weiden, they were the first to recognize the graft-versus-leukemia effect. This mechanism is responsible for decreasing the likelihood of leukemia relapse following transplant and has paved the way for modern immunotherapies.

One of Dr. Storb’s most notable accomplishments is the development of the non-myeloablative conditioning regimen and transplant, sometimes called the “mini-transplant.” It involves minimal pre-transplant radiation, is less intense than standard transplantation, and depends on the graft-versus-tumor effect. These innovations expanded access to transplants, enabling older and medically frail patients to undergo the procedure.

ASH MENTOR AWARDS
ASH will recognize Jorge Di Paola, MD, of the Washington University in St. Louis, for his role in helping teach and inspire the next generation of pediatric classical hematologists. Dr. Di Paola leads his trainees with an inclusive and nurturing spirit. He credits his mentors for shaping and supporting him on his journey from medical school in Argentina to residency, fellowship, and research in the United States, and he brings that experience to his mentees. Dr. Di Paola’s genuine investment in his trainees’ professional and personal success, coupled with a commitment to scientific excellence, has propelled dozens of young clinician-scientists on the path to success.

ASH will recognize Sophie Lanzkron, MD, MHS, of Thomas Jefferson University, for providing hands-on, lifelong mentorship to physician-scientists passionate about improving care for patients, especially those living with sickle cell disease. Her guidance is tailored, academically challenging, and validating, with many trainees crediting her for helping them navigate imposter syndrome. Dr. Lanzkron has served as a vital role model to a pool of mentees from numerous backgrounds and specialties. Her emphasis on taking risks, pivoting from failure, pursuing new opportunities, and prioritizing work-life balance has helped her mentees realize and act on their potential, establish fulfilling careers, and lead change in the field.

ASH ADVANCING INCLUSIVE EXCELLENCE AWARD
ASH will recognize Diane Krause, MD, PhD, of Yale School of Medicine, for her commitment to removing barriers that prevent the full participation of all who are interested in hematology. She has made meaningful contributions at the individual, institutional, and systemic levels by recruiting, supporting, and championing individuals from a wide range of backgrounds, including - but not limited to - those identifying as LGBTQ+ and those facing social or economic disadvantage. Dr. Krause leverages her own experiences to create an environment where all trainees feel safe, valued, and respected, better enabling the next generation of hematologists. She believes that scientific innovation depends on creativity, which is enhanced when people of different backgrounds and viewpoints work together.

Chair:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

Announcement of Awards: William Dameshek Prize and Henry M. Stratton Medals

WILLIAM DAMESHEK PRIZE

ASH will recognize Mark Dawson, MD, PhD, of Peter MacCallum Cancer Centre, with the William Dameshek Prize for his contributions to the understanding of epigenetics, the process by which our DNA is accessed to turn genes on and off, and its relationship to the development of hematologic cancers. His lab is credited with identifying how various epigenetic proteins, including BRD4 and KAT7, influence gene expression to maintain cancers such as leukemia and lymphoma. He has pioneered molecular insights into how cancers co-opt the activity of epigenetic proteins to evade recognition and eradication by the immune system. Dr. Dawson is a leader in the new and rapidly expanding field of cancer cell plasticity, whereby cancer cells use epigenetic mechanisms to rapidly adapt to cancer therapies. His discoveries have shaped how hematologists think about epigenetic regulation of hematologic malignancies and have provided the field with novel epigenetic therapies and cutting-edge strategies to improve clinical outcomes for patients with blood cancers.

HENRY M. STRATTON MEDAL

ASH will recognize Karina Yazdanbakhsh, PhD, of New York Blood Center Enterprises, with the Medal for basic science for her outstanding research in transfusion medicine, which has led to advances in the understanding and treatment of several classical (non-malignant) blood disorders. Her research explores the interplay of the immune system with platelets or red blood cells in blood disorders, including in the context of transfusions, where they can lead to immune-mediated complications. Dr. Yazdanbakhsh’s research into the patterns of immune dysregulation - which increases susceptibility to illness, infection, and inflammation - in patients living with sickle cell disease is providing the foundation for more targeted, effective therapies, while informing strategies to mitigate immunologic adverse responses to blood products and improve their transfusion management. Her contributions to the field extend beyond scientific research; Dr. Yazdanbakhsh also serves as a dedicated advocate, leader, and mentor.

ASH will recognize David A. Williams, MD, of Boston Children’s Hospital and Dana-Farber Cancer Institute, Harvard Medical School, with the Medal for clinical science for his vital contributions to the understanding of hematopoietic stem cells and viral vectors, which have led to landmark clinical trials of gene therapy for inherited blood disorders. Dr. Williams is a former ASH president and pioneer of gene therapy, having been the first person to demonstrate gene transfer into hematopoietic stem cells in mouse models using retrovirus vectors, and his research laid the groundwork for rFN-CH-296 (RetroNectin), a standard reagent used in gene transfer. Dr. Williams’ work directly enabled the development of groundbreaking gene therapies for severe combined immunodeficiency, chronic granulomatous disease, cerebral adrenoleukodystrophy, and sickle cell disease, offering additional therapy options and renewed hope for patients living with these debilitating blood disorders. His laboratory focuses on GTPases, proteins that play a crucial role in cellular processes, and identified the molecular causes of three different rare hematologic conditions due to mutations of RAC2, RHOH, and SEPTIN6.

Chair:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

ASH-EHA Joint Symposium - Menin Inhibitors: Novel Targeted Agents for Treatment of Acute Myeloid Leukemia

The ASH-EHA Joint Symposium addresses global issues in hematology and provides insight on how international collaboration can help address these issues. This year marks the 20th anniversary of this shared symposia.

This year's topic is Menin Inhibitors: Novel targeted agents for treatment of AML. AML is an aggressive heterogeneous disease with limited treatment options. Although most patients with AML receiving intensive chemotherapy can achieve a CR, the majority will relapse with 5-year OS (without HCT) being 25%. Between 1973 and 2017, no new drugs were approved for treatment of AML. The identification of unique gene expression profiles in AML has spurred significant advances in our understanding of AML and led to development and FDA approval of 12 novel agents since 2017 that target key genetic drivers of specific subtypes of AML. Menin is a member of the KMT2A (lysine methyl transferase 2A, formerly known as MLL1) complex that occupies a broad region around the promoters of a small subset of genes involved in development and cell cycle control. Menin target genes can be essential dependencies for solid and hematopoietic malignancies, especially in rearranged KMT2A (KMT2Ar) and NPM1-mutated AML, in which a stemness transcriptional program involving HOXA/MEIS1 is activated to induce malignant transformation. Small molecule inhibitors of menin have emerged as promising new agents for KMT2Ar or NPM1-mutated AML, and more recently for NUP98 fusions. A total of 7 different menin inhibitors have been developed with 2 already granted breakthrough therapy designation by the FDA. However, the mechanisms that determine menin-KMT2A occupancy and sensitivity to menin inhibition remain unknown. This session will focus on the rational design and development of menin inhibitors, their mechanisms of action and resistance mechanisms, clinical efficacy, safety, use in combination therapies, biomarkers of response, and appropriate molecular tests to detect MRD. It will also highlight the application of integrated multiomic platforms including CRISPR screens, proteogenomics, transcriptomics, and single cell analyses.

Co-Chairs:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

Konstanze Döhner
Ulm University
Ulm, Ulm, Germany

Best of ASH

Best of ASH (BOA) highlights the most cutting-edge science presented in the oral abstract sessions during the Annual Meeting. This year’s session will consist of talks arranged in arranged in overarching themes and will be presented by the 2025 Scientific Program Co-Chairs: Drs. Betty Pace (Augusta University) and Esther Obeng (St. Jude Children's Hospital).

Co-Chairs:

Betty Pace

Esther Obeng

E. Donnall Thomas Lecture and Prize

Dr. Nancy Speck, a renowned researcher, is being honored for her pivotal work in hematopoiesis and leukemogenesis. Her discovery of the transcription factor complex “core binding factor” has enabled significant conceptual insights into embryonic blood cell formation. One subunit of core binding factor is the transcription factor RUNX1, encoded by a gene responsible for blood cell creation. This factor is mutated in individuals with familial platelet disorder with associated myeloid malignancy - a condition that predisposes patients to developing myelodysplastic syndromes and leukemia. Dr. Speck is a first-generation college graduate whose decades of research have had a profound impact on both classical and malignant hematology.

Chair:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

Speaker:

Nancy Speck
University of Pennsylvania
Philadelphia, PA, United States
Paradigm-Shifting RUNX1 Science

Ernest Beutler Lecture and Prize

This lecture and prize recognizes the exceptional contributions of Drs. Radek Skoda and Ruben Mesa to the understanding and treatment of myeloproliferative neoplasms (MPNs). Dr. Skoda was instrumental in identifying the genetic basis for MPNs, while Dr. Mesa significantly advanced the development and evaluation of several MPN therapies.

Dr. Skoda, the recipient of the Ernest Beutler Lecture and Prize in basic science, is being honored for his role in discovering a frequently occurring mutation in the JAK2 gene and providing mechanistic insights into the development of MPNs. Under his leadership, Dr. Skoda’s research team at the University Hospital Basel in Switzerland determined that the mutation leads to overactivity of the JAK2 protein in a single blood stem cell, allowing it to divide rapidly and displace normal blood cells. His work provided the foundation for developing JAK2 inhibitors - therapies used to manage symptoms in individuals with MPNs.

Dr. Mesa, the recipient of the Ernest Beutler Lecture and Prize in clinical science, is being recognized for his essential work in clinical trials of JAK2 inhibitors, including those for ruxolitinib, the first JAK2 inhibitor approved by the U.S. Food and Drug Administration (FDA) for myelofibrosis. He led the development of two standardized tools for assessing symptoms in patients with MPNs: the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and the Myelofibrosis Symptom Assessment Form (MSAF). These patient-reported outcome measures were critical in supporting the approvals of multiple JAK2 inhibitor therapies and have become the gold standard for patient-reported outcome assessments in drug development across medical disciplines.

Together, the work of Drs. Skoda and Mesa has paved the way for the development of four FDA-approved JAK2 inhibitor therapies for MPNs, offering patients reduced symptoms and a better quality of life.

Chair:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

Speaker:

Radek Skoda
University Hospital Basel
Basel, Switzerland
Basic Science

Ham-Wasserman Lecture

The Ham-Wasserman Lecture was named in honor of two past presidents of ASH, Thomas Hale Ham and Louis R. Wasserman, distinguished hematologists who contributed extensively to the Society. The Ham-Wasserman lecture is traditionally given by an individual from outside the United States who has made a major contribution to our understanding of an area that relates to hematology.

Chair:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

Speaker:

Chiara Bonini
OSPEDALE SAN RAFFAELE
Milano, Italy
Gene Transfer and Genome Editing of T Cells for Cancer Immunotherapy

Presidential Symposium

Diverse hematopoietic stem and progenitor cell (HSPC) clones contribute to normal hematopoiesis. Aging is associated with the acquisition of myeloid malignancy-associated mutations that confer a fitness advantage to some of the clones and are detectable in the peripheral blood of otherwise healthy individuals. Those with detectable clonal hematopoiesis (CH) of indeterminate potential (CHIP), which occurs in 10-20% of people >70 years old, have an increased risk of myeloid malignancy associated with a single nucleotide variant > or = to 2% in the peripheral blood and a lifetime risk of developing a myeloid malignancy as well as risks of developing atherosclerotic cardiovascular disease (ASCVD), stroke, chronic liver disease, gout, CKD, COPD, and heart failure. CHIP has also been associated with lung and breast cancer. Although CHIP was described just 10 years ago, improved technologies have led to its increasing recognition and association with poor survival upon malignant transformation. The mechanisms that promote malignant transformation of CHIP are poorly understood, underscoring the urgent need for additional studies and identification of molecules (natural and synthetic) that affect gene expression, chromatin accessibility, and cytokine/chemokine receptor expression and signaling. Both cell-intrinsic and extrinsic processes appear to be involved and are context-dependent. An intergenic variant near TET2 has been observed in individuals of African ancestry that confers a 2.4-fold increase of CHIP. Relatives of patients with MPNs have a 6-8-fold increase in developing an MPN. The scarcity of detailed published data regarding germline predisposition (BMFs, telomeres, ETV6 variants, etc.) underscores the need for additional investigation for proper genetic counseling, surveillance, and personalized medicine. New analytic platforms combining single cell sequencing, epigenomics, spatial transcriptomics, proteomics, and synthetic chemistry along with flow cytometry, mass spectrometry, and functional assays will be critical to unraveling CH, CHIP, and malignant transformation.

Dr. Churpek will describe the currently known hereditary hematologic malignancy predisposition syndromes that increase risk for myeloid malignancies. She will illustrate how individual syndromes and their causative genetic variants reveal insights into mechanisms governing normal hematopoiesis. Further, she will summarize the growing body of literature that has begun to elucidate the prevalence and patterns of syndrome-specific clonal hematopoiesis. She will illustrate how specific clonal hematopoiesis variants can serve as proof of variant pathogenicity and at the same time reveal how different strategies to overcome these specific blocks in hematopoiesis either rescue the defect without (i.e., adaptively) or with (i.e., maladaptively) increasing risk for myeloid malignancies.

Dr. Pietras will address the intersection between mitochondria, metabolism, and inflammation as a driver of HSPC selection in the context of CH and discuss potential novel approaches or therapies to prevent malignant transformation. He will also discuss the roles of Cul5, polyphenols, and the microbiome in CHIP.

Dr. DiPersio will discuss the synergistic effects of combining novel CXCR4 and VLA-4 inhibitors for stem cell mobilization and ex vivo gene therapy and the use of JAK inhibitors to reduce GVHD and promote donor allogeneic HSC engraftment across major MHC barriers after antibody-based conditioning regimens. Finally, Dr. DiPersio will address the need for prospective, well-controlled studies to investigate new strategies for risk-adapted prophylaxis and salvage therapies for post-transplant relapse.

Chair:

Belinda Avalos
Atrium Health
Charlotte, NC, United States

Speakers:

Jane Churpek
University of Wisconsin-Madison
Madison, WI, United States
Germline Predisposition for Myeloid Neoplasms

Eric Pietras
University of Colorado
Aurora, CO, United States
Role of the Microenvironment in Leukemogenesis

John DiPersio
Washington University School of Medicine
Saint Louis, MO, United States
Novel Therapeutic Approaches