The content of this continuing medical education (CME) activity is derived from a symposium, “The Promise of Epigenetic Therapy,” presented at the 48th Annual Meeting of the American Society of Hematology.
The myelodysplastic syndromes (MDS) have often been called the poster child for apoptosis gone awry: in some cases death (to hematopoietic progenitors) comes too infrequently, in other cases it comes too readily. The quest to better understand its basis and to translate this understanding into therapeutics is only beginning, but already several therapies have emerged and others are in development. For now, there is only one curative therapy for MDS: hematopoietic cell transplantation (HCT).
Management of myelodysplastic syndrome has been disappointing for years. The mainstay approach was only supportive care. A malaise settled all around. Today there are new Food and Drug Administration cleared therapies, and novel agents are on the horizon. True progress has been small to date, but with the number of new therapies and new classes of agents undergoing study, the future is hopeful.
Try as hard as it can, the myelodysplastic bone marrow just cannot seem to meet the ordinary day-to-day needs for blood cells. Indeed, failure is destined no matter what we do. Then, when it seems as bad as it can get, it only gets worse: total marrow shutdown or change into leukemia. Effective therapy has been long overdue.