Myelodysplastic Syndrome (MDS) Clinical Trials & Research

Researchers are continually testing new treatments for MDS. Many of these studies originate at Memorial Sloan Kettering. The clinical trial process is designed to advance the current standard of care for MDS.

Our doctors have led many of the clinical trials for drugs that are now considered part of standard care for MDS. In this same way, we hope to develop better treatments for MDS in the future.

As with all cancer treatment at Memorial Sloan Kettering, clinical trials involve a team approach. Doctors, scientists, and pathologists work together to care for each person with MDS. We are experts at determining who will have the most benefit from which trial.

We routinely conduct a detailed genetic analysis on the bone marrow samples from all people who are newly diagnosed with MDS. This helps us fine-tune your prognosis and identify personalized treatments that are tailored to your disease. For example, if you have a mutation in one of the so-called splicing genes, such as SF3B1 or SRSF2, you may be a candidate for our ongoing clinical trial of a novel splicing-modulator drug called H3B-8800. It was developed in part by scientists at MSK.

Here you can find a continually updated listing of Memorial Sloan Kettering’s current clinical trials for MDS.

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10 Clinical Trials found
Researchers want to find the best dose of BH-30236 to treat people with leukemia. This study includes people with acute myelogenous leukemia (AML) that relapsed (came back) after treatment. It also includes people with higher-risk myelodysplastic syndrome (MDS). Higher-risk means a disease has a higher chance of coming back or not responding to standard treatment.
Researchers want to find the best dose of CBX-250 to treat leukemia that came back or keeps growing after treatment. The people in this study have one of these diseases:
Researchers are seeking the best dose of cirtuvivint to use alone and with ASTX727 for leukemia and myelodysplastic syndromes (MDS). The people in this study have acute myeloid leukemia (AML) or MDS that keeps growing after treatment.
In this study, researchers want to find the best dose of REM-422 to treat leukemia or myelodysplastic syndrome (MDS). The people in this study have acute myeloid leukemia (AML) or higher-risk MDS that keeps growing even with treatment. Higher-risk means the disease has a high chance of coming back or not responding to therapy.
Graft-versus-host disease (GVHD) is a serious condition that can happen after a stem cell transplant from a donor. The donated cells see the healthy tissues in the recipient's body as foreign and attack them. TRX103 is a new drug designed to reduce the risk of GVHD.
Researchers want to find the best dose of eganelisib when used alone and with cytarabine to treat leukemia. The people in this study have acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS) that has come back or keeps growing after treatment.          
Researchers want to see if ASTX727 is practical and safe for people with high-risk clonal cytopenia of undetermined significance (CCUS). With CCUS, there is a mutation (change) in one or more of the genes that help blood cells develop. People with CCUS have low levels of certain kinds of blood cells. CCUS can lead to precancerous conditions such as myelodysplastic syndrome or blood cancers such as acute myeloid leukemia.
The transplantation of stem cells from umbilical cord blood is a treatment for some blood cancers and non-cancerous blood or metabolic disorders. Patients routinely receive high doses of chemotherapy and sometimes radiation before receiving the stem cells to help make room in the bone marrow for new blood stem cells to grow, prevent the body from rejecting the transplanted cells, and help kill any abnormal blood cells in the body. However, the combination of these treatments can have serious side effects.
Umbilical card blood is rich in healthy, blood-forming cells (stem cells) that are very good at fighting blood cancers. Cord blood transplants (CBT) are a standard treatment for adults with blood cancers who lack a genetically matched stem cell donor. Cord blood is donated by a baby's mother at birth. CBT uses stem cells from cord blood collections to replace cells that have been destroyed by disease or anti-cancer treatment.