A cytoprotective agent or chemoprotectant. This drug is approved by the U.S. Food and Drug Administration (FDA) for use during cancer therapy to protect the body's normal cells. Amifostine is being studied in clinical trials, as a single agent or in combination with other medicines, for treating myelodysplastic syndromes.

ATG is an immunosuppressant, a drug that lowers the body's immune response. Scientists believe that aplastic anemia happens when the immune system attacks and destroys bone marrow stem cells. ATG kills the specific cells that are attacking the bone marrow stem cells. This allows the bone marrow to grow and make new blood cells. ATG may be used to treat other bone marrow failure diseases in some cases. ATG is approved by the U.S. Food and Drug Administration (FDA) for treating moderate and severe aplastic anemia. ATG is commonly used with another drug called cyclosporine.

An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells. It is approved by the U.S. Food and Drug Administration (FDA) for treating acute promylocitic leukemia (APL). Currently, researchers are studying arsenic trioxide, as a single agent or in combination with other medicines, for treating myelodysplastic syndromes.

A demethylating agent, this medicine interacts with DNA to restore normal growth of blood cells in the bone marrow. It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U.S. Food and Drug Administration (FDA) for treating all myelodysplastic syndromes (MDS) subtypes.

An immunosuppressant, this drug that lowers the body's immune response. Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure.

A growth factor (cytokine), it is a man-made version of a naturally occurring substance in the body, called erythropoietin, that encourages the bone marrow to make more red blood cells. It is in a class of medicines called erythropoiesis-stimulating agents (ESAs). Darbepoetin alfa can help improve red blood cell counts in bone marrow failure patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous). Darbepoetin alfa is approved by the U.S. Food and Drug Administration (FDA) for treating anemia.

A demethylating agent, this drug interacts with DNA to restore normal growth of blood cells in the bone marrow. It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Decitabine is approved by the U.S. Food and Drug Administration (FDA) for treating all MDS subtypes, though it is used most commonly to treat higher-risk MDS.

A man-made version of a naturally occurring substance in the body (erythropoietin) that encourages the bone marrow to make more red blood cells. Epoetin alfa can help improve red blood cell counts in bone marrow failure disease patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous). Epoetin alfa is approved by the U.S. Food and Drug Administration (FDA) for treating anemia. It is in a class of drugs called growth factors (cytokines).

A man-made version of a naturally occurring substance in the body that promotes white blood cell growth in the bone marrow. It is known as a hematopoietic growth factor or colony stimulating factorand is given by injection under the skin (subcutaneous) or in the vein (intravenous). Filgrastim is approved by the U.S. Food and Drug Administration (FDA) for use in cancer patients who are at risk of developing infection due to cancer treatment. It is also approved for treating other patients who have a very low white blood cell count. It is in a class of drugs called growth factors (cytokines).

An immunomodulatory and anti-angiogenic agent, this drug slows the growth of blood vessels feeding abnormal cells and kills abnormal cells in the bone marrow. 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 patients, lenalidomide can improve anemia and reduce or eliminate the need for blood transfusions.

A drug that lowers the body's immune response, also referred to as an immunosuppressantdrug. Mycophenolate mofetil is approved by the U.S. Food and Drug Administration (FDA) to prevent organ rejection in people who have received a kidney, heart, or liver transplant. This medicine comes as a capsule, tablet, or liquid that is taken by mouth. Researchers are currently studying MMF in combination with other medicines for treating aplastic anemia.

R115777, a farnesyl transferase inhibitor, blocks naturally occuring enzymes from activating harmful proteins, which occurs in roughly 25% of myelodysplastic syndromes (MDS) patients.

A man-made version of a naturally occurring substance in the body that promotes white blood cell growth in the bone marrow. It is known as a hematopoietic growth factor or colony stimulating factorand is given by injection under the skin (subcutaneous) or in the vein (intravenous). Filgrastim is approved by the U.S. Food and Drug Administration (FDA) for use in cancer patients who are at risk of developing infection due to cancer treatment. It is also approved for treating other patients who have a very low white blood cell count. It is in a class of drugs called growth factors (cytokines).

TLK-199 is a small molecule drug that stimulates the bone marrow to produce blood cells. Currently TLK-199 is in Phase II of the Food and Drug Administration’s (FDA’s) clinical trial for the treatment of myelodsyplasitc syndromes (MDS).

Thalidomide (Thalomid) is an old drug, though it has not been widely available since the 1960s, when it was found to cause birth defects. Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy. Thalidomide is being studied, either as a single agent or in combination with other medicines, for treating of myelodysplastic syndromes (MDS).

VNP4010M is a sulfonylhydrazine agent, which works by damaging DNA. It is currently in Phase II of the Food and Drug Adminstration's clinical trials for treatment of myelodysplastic syndromes (MDS) patients.