Scientific Committee on Bone Marrow Failure
Jean Soulier, MD ('22)
Marcin Wlodarski, MD, PhD ('22)
Daria V. Babushok, MD ('23)
Luis Batista, PhD ('24)
Carlo Dufour, MD ('23)
Austin Kulasekararaj, MD, MBBS, FRCPath, MRCP ('25)
Usua Oyarbide, PhD ('25)
Sharon A. Savage, MD ('24)
Hannah Tamary, MD ('22)
Adrianna Vlachos, MD ('24)
The Scientific Committee on Bone Marrow Failure’s major focus is on all issues related to acquired and inherited bone marrow failure (BMF). Until recently, the BMF syndromes were an unexplained mélange of conditions, some inherited, some sporadic, some acquired, all characterized by a failure of blood cell production that may affect one, several or all blood cell lineages. Research in the field of BMF lagged behind mainly due to the fact that the tissues affected and the cells to be studied are, by definition, missing. However recent advances, particularly in genetics, led to a more accurate definition of the various forms of BMF and provided insights into disease pathology. These insights were frequently unexpected and uncovered novel biological principals with implications reaching far beyond the individual disease pathology. Examples are impaired telomere maintenance in Dyskeratosis Congenita and its implications in aging, DNA repair in Fanconi Anemia and its implications in cancer and cancer treatment, ribosome biogenesis and its implication for cell growth and cell division and the pathways of the immune system to cause or maintain acquired forms BMF. With the additional development of new tools modeling the disease pathology and the ability to specifically design or screen for new drugs a new and fast evolving discipline in Hematology has emerged that needs the patronage and mentorship of the ASH community in order to support teaching and education in BMF, to stimulate basic, translational and clinical research, to assist in the lobbying for research funding, and to help in establishing multi-institutional collaborations, nationwide patient registries, patient sample repositories, and multi-institutional clinical trials.
The interest of the Scientific Committee for Bone Marrow Failure covers clinical and basic science aspects including the investigation of disease causes and mechanisms, the identification and evaluation of diagnostic targets and tools, the discovery, assessment and modus operandi of novel therapeutics as well as the study of late effects caused by the disease mechanisms or the treatment thereof.
There are potential scientific overlaps with areas covered by several other scientific subcommittees, these include Scientific Committee on Blood Disorders in Childhood, Scientific Committee on Hemoglobin/Red Cell, Scientific Committee on Myeloid Biology, Scientific Committee on Myeloid Neoplasia, Scientific Committee on Platelets, and the Scientific Committee on Transplantation Biology. This is because many of the leading laboratories in BMF are currently represented in these scientific subcommittees. Although this enriches the spectrum of those subcommittees, and is likely to continue to do so, as a new emerging discipline in benign hematology the Scientific Committee on Bone Marrow Failure will need to bring together expertise from all of those disciplines to focus on the major issues relevant to BMF and thereby facilitate the translation of new discoveries and technologies directly to the bedside of patients with BMF. In contrast to above mentioned scientific committees, the Scientific Committee for Bone Marrow Failure will have a strong emphasis on the deranged pathways that are responsible for disease, and those that determine disease outcome, late complications and response to treatment. In this regard the Scientific Committee for Bone Marrow Failure will be complementary rather than competitive with other existing ASH scientific subcommittees. Furthermore the Scientific Committee for Bone Marrow Failure will advise ASH on questions of education and teaching in BMF diseases, as well as assist in establishing guidelines for standardization of diagnosis, prognostic indicators, treatment approaches and response to treatment. Finally, the Scientific Committee for Bone Marrow Failure will assist and consult ASH in its advocacy efforts for research support and in ventures to facilitate nationwide registries and bio- and data- repositories as well as the globalization of established protocols.