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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

Researchers 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 clear that not everyone with MDS has the same gene changes in their cancer cells. An improved understanding of this is helping to better classify different types of MDS and to determine a person’s likely prognosis (outlook). It’s also helping doctors determine which patients might benefit most from different types of treatment.

The US National Cancer Institute (NCI) is now running a series of clinical trials, known as MyeloMATCH. People who join MyeloMATCH have their blood and bone marrow tested for certain gene changes on the cancer cells. A person may then be eligible for a clinical trial studying a treatment that’s tailored to the changes found in their cells.

Newer types of molecular lab tests are also becoming more important in the treatment of MDS. For example, if a person’s MDS progresses, doctors often look at the MDS cells to see how they’ve changed. Sometimes new gene changes have appeared in the cells, which might make them more likely to respond to newer types of targeted drugs.

Chemotherapy

Drugs called hypomethylating agents, such as azacitidine and decitabine, are currently some of the most effective drugs in treating MDS. But unfortunately, they’re not helpful for everyone, and over time they often stop working.

Researchers are now looking for new chemo drugs and drug combinations that might work better, as well as have fewer side effects.

Researchers are also testing oral (by mouth) forms of these drugs, which might be easier for patients to take. One of these, Inqovi (decitabine with cedazuridine), is already being used.

Targeted therapy drugs

Targeted 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 chemo doesn’t, and they tend to have different (and sometimes less severe) side effects.

As researchers have learned more about the gene changes inside MDS cells, they’ve started to test drugs that target some of these changes. An example is ivosidenib (Tibsovo), which can now be used to treat MDS when the cells have an IDH1 gene mutation.

Many other targeted therapy drugs are now being studied for use against MDS as well.

More general information on this type of treatment can be found in Targeted Therapy.

Stem cell transplant

At this time, a stem cell transplant (also known as a bone marrow transplant) offers the best chance for long-term remission (and a possible cure) for people with MDS. But this is an intense treatment, and many people with MDS can’t tolerate it.

Researchers continue to look for ways to make this procedure more effective, reduce complications from it, and possibly make more people eligible to be helped by this treatment.