What's New in Myelodysplastic Syndrome Research and Treatment?

Genetics and biology of MDS

Research on the causes, diagnosis, and treatment of myelodysplastic syndromes (MDS) is being done at many cancer research centers. Scientists are making progress in understanding how a series of changes in a person’s DNA can cause normal bone marrow cells to develop into myelodysplastic cells.

Scientists are also learning how bone marrow stromal cells influence MDS cells. Bone marrow stromal cells are found in the bone marrow but do not develop into blood cells. Instead, they help support, nourish, and regulate the blood-forming cells. Recent studies suggest that although the stromal cells in MDS patients are not cancerous, they are not normal either, and seem to have a role in causing MDS. Scientists have identified some of the chemical signals that are exchanged between stromal cells and MDS cells.

As more information from this research unfolds, it may be used to design new drugs or eventually in developing gene therapy. This approach replaces the abnormal DNA of cancer cells with normal DNA to restore normal control of cell growth.


Studies are being done to find drug combinations that work well without serious side effects. New drugs are continually being developed and tested. The drugs sapacitabine and clofarabine have both shown promise. Also, an oral (by mouth) form of azacitidine is being tested.

Research is also under way to see if there is a group of patients that may benefit from more intensive chemotherapy.

Immune suppression

Researchers are also looking at different ways to block patients’ immune systems. The drug alemtuzumab (Campath), which is more often used to treat lymphoma and a certain type of chronic leukemia, acts by attacking T-cells. This suppresses the immune system, and was helpful in a recent study in MDS.

Targeted therapy

Targeted therapy is a newer type of cancer treatment that uses drugs or other substances to identify and attack cancer cells while doing little damage to normal cells. These therapies attack the cancer cells’ inner workings – the programming and gene changes that make them different from normal, healthy 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.

Some targeted therapy drugs, called angiogenesis inhibitors, work by preventing growth of new blood vessels. This type of drug has been helpful in treating some types of cancer that form tumors, but may also be helpful in cancers like leukemia and MDS that grow in the bone marrow. Other types of targeted therapy drugs target certain abnormal genes in cancer cells. Some drugs that have been studied in MDS include bevacizumab, aflibercept, everolimus, sorafenib, sunitinib, and midostaurin.

More information can be found in Targeted Therapy.

Stem cell transplant

Scientists continue to refine this procedure to increase its effectiveness, reduce complications, and determine which patients are likely to be helped by this treatment.

Drugs to help blood counts

Romiplostim (Nplate®) is a new drug that raises platelet counts. It is approved to treat patients who have a disease (called ITP) 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.

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: February 10, 2014 Last Revised: July 2, 2015

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