Immunotherapies- Chimeric antigen receptor T-cell (CAR-T) therapy, checkpoint blockade, bispecific antibodies, and many others — are highly successful for malignancies such as acute lymphoblastic leukemia, diffuse large B-cell lymphoma, and multiple myeloma, resulting in numerous drug approvals. Immune-based therapies have also been remarkably useful in classical hematologic diseases such as hemophilia and other coagulopathies (novel bispecific antibodies), bone marrow failure (immunosuppressive biologics and drugs), and complementmediated diseases like paroxysmal nocturnal hemoglobuinuria (C5 and other blockers).
Despite these accomplishments, longterm benefit is limited by durability of response and is dependent on numerous emerging factors that vary depending on the type of immunotherapy, including toxicity profile, tumor mutational burden, and disease burden. There is also a rapidly evolving regulatory landscape, particularly for treatments that involve genetic manipulation, as more of these therapies reach clinical trials.
Improvement of immune-based therapies for the treatment of hematologic diseases is one of ASH’s research priorities and is a major focus of ASH’s Precision Medicine Initiative, led partly by the ASH Subcommittee on Emerging Gene and Cell Therapies. To examine preclinical and clinical factors influencing the effective development, regulation, and implementation of immunotherapies for hematologic diseases, the Subcommittee (then a Task Force) organized a Summit on Emerging Immunotherapies for Hematologic Diseases in the summer of 2018. The meeting addressed the current state of the science in immunotherapies and discussed critical gaps in the field.
Building on the scientific discussions that began at the 2018 summit, ASH is now hosting a 2023 Summit on Immunotherapies for Hematologic Diseases on March 2-3 in Washington, DC. This in-person event will be a unique meeting of the minds among the academic, medical, industry, and federal regulatory communities to address the obstacles in immunotherapy treatment development for both classical and malignant hematologic conditions.
The summit will begin with a keynote address by Dr. Stanley Riddell from the Fred Hutchinson Cancer Research Center, one of the pioneers in cellular immunotherapy, followed by a discussion of new insights into the science of immunotherapy. The past four years have seen multiple approvals of CAR-T therapies and bispecific antibodies for hematologic malignancies resulting in a large increase in real-world experience.
To address vital contemporary needs, summit sessions will discuss critical limitations of immunotherapies and gene therapy including associated adverse events, as well as examine cellular and molecular mechanisms involved in resistance and relapse associated with these therapies. Two sessions will focus specifically on lessons learned from the clinical experience with immunotherapies in hematologic diseases. A third session will discuss the spectrum of unique toxicities associated with these therapies, with an emphasis on biomarkers, prediction algorithms, and management. Clinical experience and emerging science have identified numerous limitations of these therapies, which will be the topic of one session, followed by a session on potential approaches to improve therapeutic efficacy.
Manufacturing of cellular immunotherapies is complex and evolving rapidly. Thus, the summit will include a session focused on manufacturing considerations from the academic, industry, and regulatory perspectives with an emphasis on the transition from early-phase clinical trial manufacturing to commercial manufacturing. The meeting will conclude with a late-breaking session (a collaborative effort between ASH and the Society for Immunotherapy of Cancer) focused on the development and application of gene editing and gene therapy for both classical hematologic conditions and malignant hematology. All sessions will include talks by field-leading experts as well as brief presentations from select submitted abstracts that will emphasize work by young investigators.
The Summit on Immunotherapies for Hematologic Diseases will convene pioneers in immune and cellular therapies, experts in hematologic diseases, and the next generation of these fields in an interactive program aimed at fostering collaboration and innovation. Visit www.hematology.org/meetings/summit-on-emergingimmunotherapies-for-hematologic-diseases to learn more and to submit an abstract by January 5, 2023.
Acknowledgment: Dr. Fry is grateful for the input of summit cochairs Shannon Maude, MD, PhD (Children’s Hospital of Philadelphia), and Neal Young, MD (National Institutes of Health).
Terry Fry, MD, is Professor of Pediatrics, Hematology and Immunology; Co-Director, Human Immunology and
Immunotherapy Initiative and Director of Cancer Immunotherapy, University of Colorado School of Medicine, Denver
