FY 2004 Testimony of the American Society of Hematology
to the House Appropriations Subcommittee on Labor, HHS, and Education
May 22, 2003

Chairman Regula and members of the Subcommittee, the American Society of Hematology (ASH) thanks you for the opportunity to submit written testimony on the fiscal year (FY) 2004 Departments of Labor, Health and Human Services and Education Appropriations. In addition, ASH sincerely thanks the Subcommittee for its unwavering support of biomedical research.

The Society represents nearly 13,000 clinicians and scientists committed to the study and treatment of blood and blood-related diseases. These diseases encompass malignant disorders such as leukemia, lymphoma, and myeloma; non-malignant conditions including anemia and hemophilia; and congenital disorders such as sickle cell anemia and thalassemia. In addition, hematologists have been pioneers in the fields of bone marrow transplantation, gene therapy, and many drugs for the prevention and treatment of heart attacks and strokes.

ASH fully supports the Ad Hoc Group for Medical Research Funding recommendation of $30 billion for NIH in FY 2004. This 10 percent increase represents the first funding level in the post-doubling era. It is essential that the nation maintain its commitment to medical research funding so that the progress made during the past five years is not eroded.

The Society is proud that NIH-sponsored research in hematology has led to important discoveries and has generated new treatments and pharmaceutical products with broad applicability to human diseases. ASH has always emphasized the synergy that is vital to successful scientific work. Basic research on the blood has aided physicians who treat patients with heart disease, strokes, end-stage renal disease, cancer and AIDS. As a result of this cross-fertilization, the Society remains firmly committed to broad-based support for biomedical research and to the existing peer-review process as the best way to identify and prioritize scientific grants.

The study of blood and its disorders involves a number of NIH Institutes, including the National Heart, Lung and Blood Institute (NHLBI), the National Cancer Institute (NCI), the National Institute of Diabetes, Digestive and Kidney Diseases (NIDDK), and the National Institute on Aging (NIA). The Society supports the leadership of these Institutes and commends them for their vision and superb stewardship.

The Society's efforts this year focus on meeting the increased clinical research opportunities in hematology in order to translate basic scientific findings into improved treatments for patients with serious blood diseases. New comprehensive approaches to clinical research funding will advance our understanding of how to treat these diseases, enable patients to participate in high quality clinical protocols, and attract and train much-needed clinicians and clinical researchers to the field of hematology.

For FY 2004, ASH seeks Congressional support for the following activities:

Enhance Support for NHLBI's Clinical Research Networks

ASH applauds NHLBI's efforts to develop clinical research networks that translate fundamental hematologic scientific advances into treatments that benefit patients with blood disorders. The Society also commends the Institute on the progress of the new network in Hemostasis/Transfusion Medicine as well as the activities of the NHLBI networks in Thalassemia, Sickle Cell Disease, and Bone Marrow Transplant Research.

The Society is particularly interested in efforts to incorporate a strong training component into the structure of these networks for beginning investigators and clinicians. Hematology faces a serious problem because an insufficient number of physician-investigators are fully trained in treating and researching complicated, chronic diseases such as sickle cell disease, hemophilia, and other bleeding and blood clotting disorders. ASH is working closely with the NHLBI leadership to effectively integrate a clinical and translational research training component into the core mission of all the NHLBI networks.

An increased emphasis on NHLBI-supported clinical research networks will expand the opportunities available for patients to participate in large, multicenter, high-quality clinical protocols. This issue is very important because hematologists treat and perform research on a large number of uncommon disorders; without strong support for the networks, researchers would not be able to arrive at statistically valid and reliable conclusions.

Increase Research into Myeloproliferative Disorders and Myelodysplastic Syndromes

Myeloproliferative disorders and myelodysplasia are two very different types of chronic diseases of bone marrow cells that have implications for the study of leukemia. ASH is very supportive of the increased level of effective collaboration between NHLBI and NCI on developing research initiatives on these diseases. In the past, research into myeloproliferative disorders and myelodysplasia has suffered because they fall between the missions of several NIH institutes.

The Society encourages NCI and NHLBI to support investigation into addressing the lack of basic knowledge about these diseases as well as developing effective treatments for them. More specifically, ASH also supports research into the vascular, thrombotic, and hemorrhagic abnormalities associated with myeloproliferative disorders and the specific abnormalities in cell gene expression that are responsible for these diseases.

With the rising incidence in the US of myelodysplasia, a blood disorder primarily of older patients and individuals who have undergone previous radiation or chemotherapy treatments for cancer, ASH seeks NCI and NHLBI support for research to better understand the causes of the disorder. As the lifespan of the average American increases, myelodysplasia-formerly a rare disorder-is becoming a much more common disease.

Current treatment for myelodysplasia is limited to blood product transfusions, and, in some cases, chemotherapy treatments if the disease develops into acute myeloid leukemia. There is no curative treatment other than bone marrow transplantation, which will benefit only a small percentage of patients. Among the victims of myelodysplasia are the astronomer and great promoter of science, Carl Sagan, and former Massachusetts Senator Paul Tsongas.

In addition, ASH favors a new research emphasis on myeloproliferative disorders, which are caused by a person's bone marrow producing too many blood cells, and may develop into acute myeloid leukemia. The myeloproliferative disorders are believed to be due to mutations that occur in adult hematopoietic stem cells that give them a growth advantage over normal blood-forming cells.

The interest and expertise of NCI in understanding the multiple steps in development of cancers, combined with the interest and expertise of NHLBI in studying normal stem cell biology, provide powerful opportunities for the development of new research initiatives. Such initiatives are likely to result in a clear understanding of the molecular mechanisms responsible for these disorders and the development of new treatments that are targeted specifically to interfere with the specific mutated proteins that cause these diseases.

Develop Proposals for Hematology-related Research into Metabolic Diseases

ASH believes that further study into the hematology-related complications of metabolic diseases, such as diabetes, is critically needed. Many of the most important manifestations of metabolic disorders are the result of microsvascular disease, including limb ischemia, vision loss, and renal failure. The Society encourages NIDDK to convene a conference that focuses on identifying specific targets for research to improve the hematologic understanding of metabolic diseases and their microvascular complications.

Boost Research Initiatives into Blood Disorders that Affect the Elderly Population

ASH encourages NIA to increase research initiatives into the basic biology and adverse quality of life complications of blood disorders in the elderly population. The frequency of many diseases increases with age and, based on projections about the expansion of the older population, there are several hematological diseases that are becoming serious health care problems.

For example, the incidence and prevalence of anemia increases with age. After age 85, one fourth of the population is anemic. For those that are anemic, there are several increased risk factors and health complications. Myelodysplasia is another disease that becomes more prevalent as one ages. The Society is intrigued by a broader research focus on hematology and aging; there are several other examples of hematologic disorders with sharply increased rates in the very elderly.

Conclusion

This is an exciting time to be engaged in biomedical research and the Society is proud that ASH members are participating in so many innovative studies. ASH praises the NIH leadership for the excellent stewardship of the hematology research portfolio at NCI, NHLBI, NIDDK, and NIA. The opportunities in hematology research are immense, particularly in translational research. Partnerships and cooperative ventures involving multiple academic centers are necessary for clinical research projects to succeed and need special attention from NIH. When properly conceived and implemented, ASH believes these studies will lead to improved therapies for patients with debilitating and deadly blood disorders.

The Society sincerely hopes that the Subcommittee will continue its longstanding support of biomedical research and will find the means to fund NIH at $30 billion in FY 2004. Thank you again for the opportunity to submit testimony. Please contact Jeff Coughlin, ASH Government Affairs Manager, at (202) 776-0544 or jcoughlin@hematology.org if you have any questions or need further information on hematology research and NIH funding.

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