The American Society of Hematology (ASH), the world's largest professional society committed to the study and treatment of blood disorders, today issued a report urging federal agencies to coordinate hematology research funding around seven specific high-need areas that would produce the greatest impact and translate into improvements in patient care in the United States.
Developed by the Society's Committee on Scientific Affairs, a team of world-class experts in hematology, the ASH Agenda for Hematology Research is a strategic plan identifying the most promising areas of research that require ongoing support.
ASH's Research Agenda illustrates how several modern hematology research breakthroughs have resulted in significant improvements in care for patients with blood disorders and cancers, such as acute lymphoblastic leukemia, chronic myeloid leukemia, and multiple myeloma. Perhaps more importantly, the ASH Research Agenda demonstrates how hematology research has led to extraordinary gains in patient care across all of medicine, and emphasizes how support for biomedical and hematology research must be protected from budget cuts because such work provides an enormous return on investment.
"Because blood runs through every organ and tissue in the body, discoveries made by hematologists about its structure and function have an enormous ripple effect that extends far beyond our discipline, to areas such as oncology, cardiology, and surgery," said ASH President Armand Keating, MD, of Princess Margaret Hospital in Toronto. "Tomorrow's hematologic discoveries are guaranteed to have a dramatic impact on the future of health in America and around the world in all areas of medicine. For that reason alone, hematology research should be placed among the top priorities in health care."
To enable future, cross-disciplinary discoveries, the Society recommends that federal agencies coordinate hematology funding to support research in the following highly promising areas of scientific investigation that are likely to produce the greatest return on investment:
- Stem Cells and Regenerative Medicine - Improve Current Technologies to Cure Blood Disorders: Accelerate efforts to bring the promise of induced pluripotent stem (iPS) cells, adult cells that have been re-programmed into an undifferentiated stem cell state and can develop into any tissue of the body, to the clinic. With sustained funding, blood stem cells could be among the first tissues to be derived from iPS cells and used clinically to treat hematologic diseases. This will set the stage for repair of other tissues and, eventually, regeneration of damaged organs.
- Myelodysplastic Syndrome and Acute Myeloid Leukemia - Find an Effective and Personalized Treatment for the Elderly: Utilize basic, translational, clinical, and public health research to explore new treatment strategies for older patients, who have not benefitted from recent successes that have led to striking improvements in outcomes for younger patients with these disorders.
- Hematopoietic Stem Cell Transplantation - Increase Success Rates by Improving Management of Graft vs. Host Disease (GVHD): Develop new models and clinical trials to explore why bone marrow or blood stem cell recipients develop GVHD, the single most important barrier to the success of these transplants, and identify biomarkers that can help risk-stratify patients, identify new treatments, and better customize current ones.
- Sickle Cell Disease - Reduce the Barriers to Care, Burden of Pain, End-Organ Injury, and Premature Death: Develop advanced, highly targeted approaches to discovery and implementation of new interventions that will improve care and quality of life for the 70,000-90,000 Americans living with this inherited disorder.
- Deep-Vein Thrombosis and Venous Thromboembolism - Understand the Risk Factors and Develop Targeted Therapies: Conduct substantial epidemiological, clinical, and basic science studies to minimize the burden of this largely under-reported, under-studied disease that has become a public health crisis in the United States, costing health-care providers more than $2 billion annually.
- Childhood Leukemia - Improve Cure Rates by Performing Coordinated Research on Novel Targeted Therapies: Design and execute coordinated research that addresses discovery of targets, preclinical testing, and clinical trials of novel targeted agents to prevent relapse and enhance survival rates of childhood and infant leukemia, a leading cause of cancer death in children.
- Translating Laboratory Advances into the Clinic - Use Novel Genomic Technologies to Improve Treatment of Hematologic Diseases: Harness recent discoveries of important genetic and biologic markers and new insights of how disease genetics and patient genetics affect response to treatment. This will help not only to develop novel therapies directed against disease targets, but also to ensure that patients receive the right treatments to maximize successful outcomes.
The ASH Research Agenda asserts that continual investment in these areas will not only yield large dividends for other disciplines, but will also collectively drive the national economy.
"A commitment to these high-yield areas will capitalize on the momentum of previous investments and will drive research progress that will develop new treatments for serious disorders, train the next generation of scientists, create jobs, and promote economic growth and innovation," said ASH Committee on Scientific Affairs Chair Robert Hromas, MD.
Ultimately, the Society believes that support of the outlined scientific priority areas will continue to propel hematology and medicine further into the personalized era.
"Insights gained from investment in these areas will uncover new genetic and biologic markers that can be used to understand what causes a disease, the risk factors that predispose to disease, and how patients will respond to a particular treatment," said Dr. Hromas, who is also Chair of the Department of Medicine at the University of Florida. "Translating these new discoveries and technologies into personalized patient care offers the possibility of better survival, less toxicity, disease prevention, improved quality of life, and lower health-care costs."
By identifying the most promising areas of research, ASH hopes to work with federal agencies and other stakeholders to prioritize and leverage limited resources with the goal of supporting research that will ultimately improve patient care.
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