UCSF Health Experts Spotlight Advances at Hematology Conference
Prestigious medical meeting features latest innovations in blood disorders and cancer
Hematologists and oncologists from around the world will present new research and clinical findings at the American Society of Hematology’s (ASH) 65th Annual Meeting and Exposition. This year’s meeting will be held in San Diego from Dec. 8-12, 2023.
Widely considered the world’s leading event in malignant and non-malignant hematology, ASH has sponsored the annual meeting for more than 60 years. The scientific program features presentations and data updates, including diagnostic developments and other advances in hematology.
This year’s scientific program highlights new and late-breaking data, including diagnostic developments by experts in multiple myeloma and other hematologic malignancies at the UCSF Helen Diller Family Comprehensive Cancer Center (HDFCCC). Here are some highlights:
Satellite Symposia: CAR T-Cell Therapies in Relapsed/Refractory Multiple Myeloma: Evaluating the Evidence for Optimal Integration
The evolution of CAR-T therapies – where do we stand now with this revolutionary treatment approach?
Friday, Dec. 8, 11 a.m. (PT)
Ajai Chari, MD, (and Sikander Ailawadhi, MD, Mayo Clinic) discuss the current role of CAR T-cell therapies in management of patients with relapsed/refractory multiple myeloma (RRMM). They will discuss and debate practical challenges such as: Is prior treatment sequencing a significant factor in patient response to CAR T-cell therapy? Should all eligible patients with RRMM receive CAR T-cell therapy, or is fitness/frailty a concern? How to prevent, identify, and manage adverse events? When is treatment discontinuation the best choice?
Session: 653. Multiple Myeloma: Prospective Therapeutic Trials: Relapsed and Refractory
Monday, Dec. 11, 4:45 p.m. (PT)
Ajai Chari, MD, will also present results from the phase 1/2 MonumenTAL-1 trial in patients with relapsed/refractory multiple myeloma (RRMM). He reports on the safety and efficacy in patients from MonumenTAL-1 who switched to less frequent or reduced dosing with tal.
Joint Session: Genome Engineering for Enhanced Blood Cancer Immunotherapy
Decoding and Rewriting Immune Cells to Advance Immune Responses to Cancer
Sunday, Dec. 10, 9:30 a.m. – 11:05 a.m. (PT)
Alexander Marson, MD, PhD, will discuss using CRISPR-mediated gene editing in primary human T cells to systematically identify genetic targets that modulate the functions of T cells in contexts ranging from immunosuppression to cancer killing. Development and application of CRISPR-based methodologies such as pooled knock-in screening, CRISPR activation, and CRISPR interference pinpoint the regulatory networks controlling T cell phenotypes and identify synthetic genetic programs to improve T cell activity in cancer, autoimmunity, and infection.
Session 634: Myeloproliferative Syndromes: Clinical and Epidemiological: Rare Myeloproliferative Neoplasms: Unveiling Promising Pathways and Novel Therapies
Saturday, Dec. 9, 9:45 a.m. (PT)
Study first author Elliot Stieglitz, MD will present the results of an open label, phase 2 trial of trametinib in children with relapsed or refractory JMML. Juvenile myelomonocytic leukemia (JMML) is an aggressive myeloproliferative neoplasm of infants and toddlers. This is the first completed study of a MEK inhibitor in any hematologic malignancy in children.
Session: 703. Cellular Immunotherapies: Basic and Translational: Overcoming Challenges in CAR-T Therapies Through Biological Insights
Saturday, Dec. 9, 10:00 a.m. (PT)
While adoptive T cell therapies have led to remarkable clinical responses in certain subsets of B cell leukemia and lymphoma, many challenges remain that are limiting the efficacy in other hematological malignancies. To identify key genes that are involved in regulating T cell dysfunction, researchers performed a genome-wide CRISPR loss-of-function screen in primary human T cells in conditions that mimic chronic antigen exposure. Study author Stefanie Bachl, MD, reports on the findings of a novel potential therapeutic target to improve T cell persistence and counteract T cell exhaustion in order to improve the efficacy of adaptive cell immunotherapies for the treatment of B-cell maturation antigen+ hematological malignancies.
Session: 703. Cellular Immunotherapies: Basic and Translational: Novel Approaches for Next Generation Cellular Immunotherapies
Oral Abstract 465: Discovery and Development of CD70 As a Cellular Therapy Target in High-Risk Multiple Myeloma
Sunday, Dec. 10, 10 a.m. (PT)
Even in the BCMA CAR-T era, multiple myeloma (MM) patients with high-risk genotypes have the poorest outcomes. In this study, researchers focused on CD70, an immunotherapeutic target known to be upregulated on many hematologic and solid tumors but minimally expressed on normal tissue. Study author Corynn Kasap, MD, PhD, reports on the study’s results which suggest cellular therapies against CD70 could be highly beneficial in high-risk MM patients, currently the greatest unmet need in the field.
Session: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Novel Therapies For Relapsed/Refractory Aggressive Lymphoma
Sunday, Dec. 10, 10:30 a.m. (PT)
AutoHCT is an effective therapy for patients with diffuse large B cell lymphoma (DLBCL) who are refractory or relapse following first-line chemotherapy, but long-term outcomes remain suboptimal. Charalambos Andreadis, MD, presents an analysis of a randomized phase III clinical trial to evaluate the addition of ibrutinib to AutoHCT as part of conditioning and as consolidation therapy for patients with relapsed/refractory ABC-DLBCL.
Session: 801. Gene Therapies: New Approaches From Bench to Bedside
Sunday, Dec. 10, 10:30 a.m. (PT)
Alpha-thalassemia major (ATM), traditionally lethal disease due to severe fetal hypoxia, is now survivable due to the advent of in utero blood transfusions. However, patients still require life-long transfusions postnatally. While allogeneic hematopoietic stem cell transplant can provide a definitive cure, it is limited by a variety of factors. Simon N Chu, MD, MS presents study findings about a CRISPR/AAV-mediated genome editing strategy to restore a full-length copy of the α-globin gene at the β-globin locus in α-thalassemia major patient-derived hematopoietic stem cells to restore normal hemoglobin production to a patient’s red blood cells.
Session: 901. Health Services and Quality Improvement – Non-Malignant Conditions: Raising the bar in Anemia, Bleeding, and Thrombotic Disorders
Sunday, Dec. 10, 5:30 p.m. (PT)
Neha Bhasin, MD, reports on the results of a prospective cross-sectional study about menstruation complications in females with sickle cell disease (SCD). Reproductive health education is lacking among individuals with SCD despite many females reporting increased pain during menstruation. The team investigated whether menstrual bleeding assessments, in the form of surveys conducted during sickle cell clinic visits, would prompt timely identification and management of menstruation complications in females with SCD.
Session: 801. Gene Therapies: The Long and the Short of Clinical Trials in Blood Disorders
Monday, Dec. 11, 5:15 p.m. (PT)
Hemophilia A is an X-linked (F8 gene) recessive disorder of hemostasis that results in insufficient factor VIII (FVIII) activity. Adeno-associated virus (AAV)–based gene therapy enables delivery of a modified F8 cDNA, allowing synthesis of functional endogenous FVIII, which prevents bleeding events. Study author Andrew D. Leavitt, MD presents updated results with nearly 4 years of follow-up on an ongoing gene therapy study in participants with severe hemophilia A.
See ASH 2023 for a complete listing of abstracts.