Myelodysplasia Real Changes…Finally

Approximately 87,000 people worldwide are diagnosed with myelodysplastic syndromes (MDS) each year, and most of us believe that the overall incidence is increasing, especially with the aging of the population. It very well may be that the incidence of MDS is very much in the running with CLL and AML to be the most common hematological malignancy in adults.

Already at this year’s meeting, several speakers, including Dr. Alan List and Dr. Michelle Le Beau, have chronicled the last two decades of change in MDS starting with the FAB classification, the International Prognostic Scoring System (IPSS), the World Health Organization’s re-classification, and now this year, finally, finally, we witnessed the FDA approval of the first drug ever specifically for MDS, that being 5-azacitidine, or Vidaza. No, it wasn’t named after the vidalia onion. But yes it really, really works. A special press session was held yesterday morning to discuss all of these recent advances, the need to increase public awareness and education, and the need for more funding to study myelodysplasia. Panelists included Dr. Steve Gore from Johns Hopkins, Dr. Eli Estey from M.D. Anderson Cancer Center, Dr. Alan List from H. Lee Moffitt Cancer Center, Dr. Stephanie Lee from Dana-Farber Cancer Institute, and myself. Yesterday and again this morning from 9:45 to 11:30 a.m., an Education Session was presented on MDS. In this session, Dr. Vardiman discusses characterization and classification of MDS and the evolution of MDS classification from the FAB system to the WHO system. The RAEBT category was eliminated in the WHO. Was it the right thing to do or not? Dr. Alan List discusses novel therapeutics for MDS, including 5-azacitidine. After many years of ho-hum we are now in the era of tremendous excitement over a very vibrant pipeline of a multitude of agents for MDS, some of them specific to MDS, others with potential applicability to other tumors. If you don’t believe me, just look at Dr. List’s Table #3 in the Education Program Book, then go to the talk to hear the real specifics. Dr. Jean-Pierre Issa discusses chromatin remodeling and epigenetic therapy in MDS. Do you understand about methylation of genes? Or maybe more appropriate to ask, do any of us really understand the ramifications of methylation and gene silencing in carcinogenesis? Believe it or not, MDS may be the disease that allows us to unravel this story. If so, it will show once again that hematologists and the study of hematological malignancies are once again blazing the trail of scientific discovery. Finally, Drs. Theo de Witte and Margriet Oosterveld discuss the role of hematopoietic stem cell transplant for MDS. When should allogeneic stem cell transplant be used? Which IPSS groups or at what disease stage should be transplanted, and what is an appropriate age cut-off? Should we T cell deplete or use reduce-intensity conditioning regimens? Is there a role for autologous transplants? What do we do about the increasing population of the very hard-to-treat patients with therapy-related MDS? Does this seem like a lot of questions? Well, that’s because we are just at the very beginning of our exploration of MDS. And to move foreward we need $$$. Luckily for us and our patients, ASH has been a major advocate in this process and now there is concrete success on that front. See the sidebar above about new $$$ from the NIH.