Aplastic Anemia & MDS International Foundation, Inc.
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Myelodysplastic Syndromes

Drugs that are considered standard and investigational therapies for myelodysplastic syndromes (MDS) are listed below, please click on their names for more information:


Generic NameBrand Name

Amifostine (Ethyol) is a cytoprotective agent or chemoprotectant, a drug approved by the U.S. Food and Drug Administration 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 the treatment of MDS.
amifostine

(Ethyol)

ATG (Atgam, Thymoglobulin) is an immunosuppressant, a drug therapy that reduces the body's natural immunity. It is approved by U.S. Food and Drug Administration (FDA) for treating moderate to severe aplastic anemia patients. ATG is most commonly used with cyclosporine, an oral medicine. ATG/cyclosporine combination is sometimes used for treating myelodysplastic syndromes (MDS), but is not nearly as effective against MDS as it is aplastic anemia.
anti-thymocyte globulin (ATG, ALG)

(Atgam, Thymoglobulin)

Arsenic trioxide (Trisenox) belongs to the general group of medicines called antineoplastics. Arsenic trioxide seems to interfere with the growth of cancer cells, which are then individually destroyed by the body. It may also affect normal cells. Researchers are studying arsenic trioxide for treating myelodysplastic syndromes (MDS) to determine whether it will be effective and safe to use.
arsenic trioxide

(Trisenox)

Azacitidine (Vidaza) is a demethylating agent, a new class of drugs. Azacitidine is thought to work by restoring normal growth and differentiation of bone marrow cells. It is approved by the F.D.A. for treating myelodysplastic syndromes.
azacitidine

(Vidaza)

Cyclosporine (Neoral, Sandimmune) is an immunosuppressant, a drug that reduces the body's natural immunity. Cyclosporine is used along with anti-thymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia.
cyclosporine

(Neoral, Sandimmune)

Darbepoetin alfa (Aranesp) promotes red blood cell production and is known as a growth factor. Growth factors are normal chemicals in the body that encourage the bone marrow to make blood cells. Growth factors have been manufactured as drugs to stimulate the bone marrow to produce more blood cells.
darbepoetin alfa

(Aranesp)

Decitabine (Dacogen) belongs to a new class of drugs called demethylating agents. Decitabine is thought to work by restoring normal growth and differentiation of bone marrow cells.
decitabine

(Dacogen)

Epoetin alfa (Epogen, Procrit) promotes red blood cell production in the bone marrow and is known as a growth factor. Growth factors are normal chemicals in the body that encourage the bone marrow to make blood cells. Growth factors have been manufactured as drugs to stimulate the bone marrow to produce more blood cells.
epoetin alfa (Erythropoietin)

(Epogen, Procrit)

Filgrastim (G-CSF) and Sargramostim (GM-CSF) promote white cell growth in the bone marrow and are known as hematopoietic (blood forming) growth factors or colony stimulating factors. They are a man-made (synthetic) version of the colony stimulating factors produced naturally in your body. These drugs are prescribed "off label" for patients with MDS and other patients with low white cell counts (neutropenia).
filgrastim (G-CSF)

(Neupoge n)

Lenalidomide (Revlimid) is a member of a new class of drugs called immunomodulatory drugs (IMiDs), drugs that can modify or regulate the functioning of the immune system. IMiDs are a group of oral drugs that are chemically similar to thalidomide. Lenalidomide is an investigative drug being studied for it's safety and effectiveness in treating myelodysplastic syndromes.
lenalidomide

(Revlimid)

Mycophenolate mofetil is an immunosuppressive agent, a drug that reduces the body's natural immunity in patients who receive organ transplants. It is currently in Phase II of the Food and Drug Adminstration's clinical trials for treatment of patients with myelodysplastic syndromes (MDS) or aplastic anemia.
mycophenolate mofetil

(Cellcept)

Prednisone, a corticosteroid, is a man-made (synthetic) version of a hormone produced naturally by your adrenal gland. It is prescribed when your body is not producing enough of this chemical on its own.
prednisone

(Zarnestra, Tipifarnib)

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

 

Filgrastim (G-CSF) and Sargramostim (GM-CSF) promote white cell growth in the bone marrow and are known as hematopoietic (blood forming) growth factors or colony stimulating factors. They are a man-made (synthetic) version of the colony stimulating factors produced naturally in your body. These drugs are prescribed "off label" for patients with MDS and other patients with low white cell counts (neutropenia).
sargramostim (GM-CSF)

(Leukine)

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).
TLK-199

(Telintra)

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).
thalidomide

(Thalomid)

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.
VNP40101M

(Cloretazine)


The information contained on these pages is not comprehensive. It should not be considered medical advice and does not take the place your physician's advice. Although AA&MDSIF strives to provide accurate and up-to-date information, it does not guarantee the accuracy or currency of this information.

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