There is a great deal of research being conducted in the area of myelodysplastic syndromes (MDS), including clinical trials and scientific studies. Areas of research for MDS include:

• Genetics. Researchers are trying to discover how changes in a person’s DNA (deoxyribonucleic acid) can cause normal bone marrow cells to develop into abnormal MDS cells. Recent studies have found a mutation of a gene known as JAK2 in many patients with myelodysplastic disorders. As more information is uncovered, researchers may eventually use their findings to develop gene therapy. This method replaces the abnormal DNA of cancer cells with normal DNA to restore normal control of cell growth.
  
  
• Stem cell transplant. Researchers are searching for ways to increase the effectiveness of stem cell transplantation, as well as reduce complications and determine which patients benefit most from the procedure.
  
  
• Chemotherapy. New chemotherapy drugs are being studied for treatment of MDS. Other studies have focused on determining the most effective combination of drugs while avoiding unnecessary side effects. In 2005, the U.S. Food and Drug Administration (FDA) approved the drug lenalidomide for individuals with certain forms of MDS. Researchers are also trying to determine whether patients with a poor prognosis may benefit from more intensive chemotherapy.
  
  
• Growth factors. Hematopoietic growth factors are hormone-like substances that stimulate blood cell production in the bone marrow. Researchers are studying growth factors that can promote the production of platelets. Additional studies are trying to determine the best method to predict which patients will benefit from growth factors.
  
  
• Differentiating agents. Differentiating agents are drugs that cause bone marrow blasts (immature cells) to develop into mature blood cells. Researchers are testing several differentiating agents including retinoids (chemicals related to vitamin A) and vitamin D. They are studying the best methods of combining these agents together or with other treatments. Arsenic trioxide is also being studied.
  
  
• Immune suppression. Researchers are studying the role of the immune system in the development of MDS. Some evidence suggests that the disease may be caused by an abnormal reaction of the immune system.
  
  
• Angiogenesis inhibitors. These drugs interfere with the growth of blood vessels. As they become available, new types of angiogenesis inhibitors are being tested for treatment of MDS.