What’s new in myelodysplastic syndrome research?
Genes and biology of MDS
Research on the causes and treatment of myelodysplastic syndromes (MDS) is being done at many cancer research centers. Scientists are making progress in learning how changes in a person’s DNA can cause normal bone marrow cells to become cancer.
Scientists are also learning how cells called stromal cells affect MDS cells. Stromal cells are found in the bone marrow, but they do not develop into blood cells. Instead, they help support, nourish, and control the blood-forming cells. Recent studies suggest that although the stromal cells in MDS patients are not cancer, they are not normal either, and they seem to have a role in causing MDS.
As more information unfolds, it might be possible in the future to use gene therapy. In this approach, the abnormal DNA of cancer cells is replaced with normal DNA to restore normal control of cell growth.
New drugs that have fewer side effects (chemo drugs as well as other kinds of drugs) are being studied. Research is also going on to see if there is a group of patients that may benefit from more intense chemo.
For some patients, blocking the patient’s immune system seems to help. The drug alemtuzumab (Campath), which suppresses the immune system, was helpful in a recent study in MDS. .
Targeted therapy is a newer type of cancer treatment that uses drugs or other substances to find and attack cancer cells while doing little damage to normal cells. Each type of targeted therapy works differently, but all alter the way a cancer cell grows, divides, repairs itself, or interacts with other cells.
Stem cell transplant
Scientists continue to refine stem cell transplants. They hope to improve how well transplant works, reduce side effects, and learn which patients are most likely to be helped by this treatment.
Drugs to help blood counts
Romiplostim (Nplate®) is a newer drug that raises platelet counts. It is approved to treat patients who have a disease in which their immune system attacks and destroys their platelets, but in more recent studies it has helped raise platelet counts in people with MDS.
Last Medical Review: 02/27/2014
Last Revised: 04/03/2014