About the ASH Agenda
The field of hematology has experienced meaningful advances in quality of care thanks to the development of novel technologies, mechanistic insights, and cutting-edge therapeutic strategies. These foundational insights are reframing modern research with the continued goal of improving outcomes and discovering cures for the most challenging hematologic diseases.
Whereas some blood disorders have benefited from 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 such as hemoglobinopathies, platelet and coagulation disorders, and orphan diseases of the hematopoietic system – continue to be associated with significant morbidity and mortality and demand attention to reduce their burden and improve quality of care worldwide.
Today, the research community looks to emerging technologies and tools in the areas of precision medicine, epigenetics, gene therapies, immunotherapy, and regenerative medicine to identify areas that have strong potential to make a dramatic impact on patient care across a range of diseases.
The ASH Agenda for Hematology Research 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 practice-changing discoveries. These specific and critically important research questions must be answered to gain insights that will launch the field into the next generation of therapies for hematologic conditions. The Agenda is updated periodically (most recently in December 2017) and is designed to be a living document. ASH encourages everyone in the hematology community to use the ASH Agenda for Hematology Research as a resource and consider citing it in publications, grant applications, or other efforts.
- Precision Medicine: Tailoring Treatment and Monitoring Response to Therapy
Enhancing precision medicine efforts in hematology will require that sequencing technologies be adopted in drug discovery efforts, and in the assessment of disease predisposition, and response to therapy. In addition, appropriate infrastructure must be developed to integrate genomic and epigenomic medicine into the clinic.
- 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.
- Stem Cell Biology and Regenerative Medicine: Development and Differentiation of 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.
- Venous Thromboembolic Disease: Opportunities to Improve Risk Prediction, Treatment, and Prevention
To improve upon the state of care for VTE, future research must aim to understand pathophysiologic mechanisms that lead to VTE in different patient populations as well as address unanswered questions about disease risk profiles, and the role of antithrombotics for VTE prevention in different clinical situations.
- 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.
- 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.