About the ASH Agenda
The field of hematology has experienced a recent surge in progress thanks to novel technologies, mechanistic insights, and cutting-edge therapeutic strategies that have driven significant and meaningful advances in the quality of care. These foundational insights are reframing modern research with the continued goal of improving outcomes and discovering cures for the most challenging hematologic diseases.
Whereas certain blood disorders have benefited from the tremendous progress in clinical research and development of new therapies, other areas have continuing challenges such as disease heterogeneity and the complex combinations of genetic drivers that have evaded effective treatment to date. A wide variety of blood-related diseases – from malignancies such as lymphoma and leukemia, to non-malignant diseases including hemoglobinopathies, blood platelet, and coagulation disorders, including orphan diseases of the hematopoietic system – continue to be associated with significant morbidity and mortality and demand attention to reduce their burden and improve the quality of care worldwide.
Today, the research community looks to emerging technologies and tools in the areas of genetics and epigenetics, gene therapies, and regenerative medicine to identify areas that have strong potential to make a dramatic impact on patient care across a range of diseases.
This Agenda serves as a roadmap for the prioritization of research support across the hematology community, including recommendations for dedicated resources from funding agencies and foundations that will equip researchers today and in the future to make truly practice-changing discoveries. These specific and critically important research questions must be answered to gain the insights that will launch the field into the next generation of care for hematologic conditions.
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- Genomic Profiling and Chemical Biology: Opportunities for Novel Targeted Therapies in Hematologic Disorders
As genomic profiling is driving significant research progress across the field, it is essential to adopt this technology in drug discovery efforts and build the infrastructure to integrate genomic medicine into the clinic.
- Genome Editing and Gene Therapy: New Opportunities to Correct Inherited Blood Disorders
While genome editing technology represents a highly promising area to advance the future of therapy for hematologic disorders, critical questions must be addressed to effectively translate this approach into clinical use.
- Epigenetic Mechanisms: Emerging Therapeutic Targets for Blood Disorders
Research on epigenetics, including its mechanisms and drivers, will elucidate highly valuable targets and lead to potentially transformative treatment regimens for hematologic disorders.
- Immunologic Treatments of Hematologic Malignancies: Moving Beyond Salvage Therapy to Curative Eradication of Minimal Residual Disease
Next-generation clinical studies will address important questions about emerging immunologic therapies but require an improved understanding of the basic biology of the immune system, including adaptive immunity, innate immunity, adjuvants, and tumor immune-surveillance.
- Stem Cell Biology and Regenerative Medicine: Reprogramming Adult Stem Cells and Differentiating Stem Cells for Replacement Tissue Products
New insights and technologies have the potential to optimize the use of stem cells and regenerative medicine, creating "designer" cells that will redefine approaches to the diagnosis and treatment of hematologic diseases.
- Preventing Venous Thromboembolic Disease: Defining Biomarkers for Prediction and Diagnosis of Recurrence
To improve upon the state of care for VTE, future research must address unanswered questions about the risk profile of this disease and the accuracy in predicting occurrence and recurrence.