New Research Shows Promise for Treating Bone Marrow Failure in MDS Patients

A study published in the August 6, 2002 edition of Annals of Internal Medicine, volume 137: 156-163, revealed that the immunosuppressive drug antithymocyte globulin (ATG) was effective in treating bone marrow failures associated with MDS. Dr. Jeffrey J. Molldrem and his colleagues at The University of Texas M.D. Anderson Cancer Center and the National Institutes of Health conducted the study. All 61 patients enrolled in the study received ATG (ATGAMñ Pharmacia Corporation) a medicine typically used in organ transplant patients. Within eight months of initial treatment, the need for red blood cell transfusions was eliminated in 21 of the 61 patients (34%) enrolled in the study. For 16 of the 21 responding patients, positive results were seen within three eight months of treatment.

On a theory that MDS has an autoimmune component, Dr. Molldrem and his colleagues elected to treat MDS as an autoimmune disease and administered ATG based on its success in treating patients with aplastic anemia, a bone marrow disorder. From 1994 to 1998, researchers enrolled MDS patients who were dependent on blood transfusions and studied their response to the drug, as well as long-term disease outcomes. Among the patients who responded to ATG, 80 percent continued making enough blood cells to be transfusion-free after five years. More importantly, none of these responding patients progressed to leukemia, a common outcome of MDS. Response was associated with a statistically significant longer survival and an almost significant decreased time to disease progression. Further research is being conducted at M.D. Anderson using other immune suppressing drugs and combinations of drugs, including ATG, to improve the response to treatment.