Advances and challenges in the treatment of myelodysplastic syndromes

Myelodysplastic syndromes Myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (MDS) is a heterogeneous group of pre-leukemic diseases characterized by peripheral blood cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. , morphologic dysplasia, and an increased risk of transformation to leukemia. MDS develop from genetically mutant clonal hematopoietic stem and progenitor cells (HSPCs) which have defects in generating mature functional blood cells due to impaired differentiation and/or survival activities. In addition, mutant HSPCs also inhibit the generation of new blood cells from remaining healthy HSPCs. Thus, the complete elimination of mutant HSPCs is the optimal goal for MDS treatment. However, most current therapies for MDS are little more than palliative, primarily addressing cytopenia-related symptoms and improving the quality of life. Only the hypomethylating agents (HMA) lenalidomide lenalidomide: Lenalidomide is a capsule that is taken by mouth. It is approved for treating low-risk, transfusion-dependent myelodysplastic syndrome (MDS) patients with an abnormality of chromosome 5q. It is currently in clinical trials to test its efficacy with a broader range of MDS patients. For some MDS… and imetelstat imetelstat: Who should not take RYTELO? Before taking RYTELO, tell your healthcare provider about all your medical conditions, including if you: are pregnant or plan to become pregnant. RYTELO may harm your unborn baby and may cause loss of pregnancy. Tell your healthcare provider right away if you… reduced the mutational burden, and then only in a small subset of cases. Many HMA-based combination therapies failed to show benefits superior to single-agent HMA treatment in clinical trials clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… . At the present time, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is still the only cure for the minority of qualified patients who have HLA HLA: See human leukocyte antigen. -matched donors. Novel effective treatments are urgently needed. Here we summarize the current standard therapeutic approaches for MDS patients and discuss major advances in MDS research and treatments. We also discuss major challenges and potential solutions to overcome these challenges for future MDS research and drug development.