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Research into the causes, diagnosis, and treatment of myelodysplastic syndromes (MDS) is being done at many cancer research centers around the world.

Genetics and biology of MDS

Scientists are making progress in understanding how changes in the DNA (genes) inside normal bone marrow cells can cause them to develop into myelodysplastic cells. It’s also clear that not all cases of MDS have the same gene changes. An improved understanding of this is helping to better classify different types of MDS and to determine a person’s likely prognosis (outlook). It might also help determine which patients might benefit most from different types of treatment.

Scientists are also learning how bone marrow stromal cells influence MDS cells. Stromal cells in the bone marrow do not develop into blood cells. Instead, they help support, nourish, and regulate the blood-forming cells. Some 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 help develop new drugs or other types of treatment.

Chemotherapy

Studies are being done to find new drugs and drug combinations that might work better, as well as having less serious side effects.

Drugs called hypomethylating agents, such as azacitidine (Vidaza) and decitabine (Dacogen), are currently some of the most effective drugs in treating MDS. But they’re not helpful for everyone, and they eventually stop working for most people. Guadecitabine is a newer drug that is related to decitabine, but it stays inside cells longer, so in theory it might work better. It has helped some people in early studies, and is now being tested in a larger study.

Researchers are also testing oral (by mouth) forms of azacitidine and decitabine, which might be easier for patients to take.

Research is also under way to see if there are some patients who might benefit from more intensive chemotherapy.

Immune suppression

In some people with MDS, the immune system seems to interfere with normal blood cell production. Some medicines, such as ATG and cyclosporine, are already being used to treat some people with MDS. Researchers are now looking at other ways to suppress the immune system in people with MDS to see if this might be helpful.

Targeted therapy

Targeted therapy drugs work differently from standard chemotherapy drugs. They affect specific parts of cancer cells that make them different from normal, healthy cells. Targeted drugs might work in some cases where chemotherapy doesn’t, and they tend to have different (and sometimes less severe) side effects. Targeted drugs are now part of the treatment for many types of cancer, and they are being studied for use in MDS as well.

For example, luspatercept is a new drug that blocks cellular proteins that are part of the TGF-beta superfamily. These proteins slow down red blood cell production. In early studies, this drug has shown a lot of promise in raising red blood cell levels in people with lower-risk forms of MDS. Further studies of this and similar drugs are under way.

Rigosertib is a new drug that targets several different proteins that normally help cancer cells grow. This drug has been shown to help some people with high-risk MDS in early studies, and is now being studied for use by itself and along with azacitidine.

Other new targeted drugs now being studied for use in MDS include:

• Imetelstat, a telomerase inhibitor
• Pevonedistat, an NAE inhibitor
• Selinexor, an XPO1 inhibitor
• Glasdegib, a smoothened (SMO) inhibitor

Many other targeted therapy drugs are now being studied as well.

More general information on this type of treatment 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 most likely to be helped by this treatment.