Skip to main content

Clinical Trials

Taking part in a clinical trial may be the best treatment choice for some MDS patients. Clinical trials are under way for all MDS-risk types. Today's standard treatments for cancer are based on earlier clinical trials. LLS continues to invest funds in MDS research.

Click here to read more about clinical trials.


Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

Current MDS Research and Clinical Trials

Below are some of the types of MDS research and trials under way:

Drug Therapy

Several combinations of FDA-approved drugs, such as azacitidine (Vidaza®) and decitabine (Dacogen®), as well as chemotherapy agents used for acute myeloid leukemia (AML) treatment, are being studied in clinical trials. Each drug works in a different way to kill cancer cells. When these drugs are used in combination, they may kill more MDS cells. Or, the drug combination may be just as effective as standard MDS therapies, but the combination drug therapy has less toxic side effects.

  • Targeted therapy. This type of treatment uses drugs or other substances to block the action of certain enzymes, proteins or other molecules involved in the growth and survival of cancer cells, but causes less harm to healthy cells
    • B-cell lymphoma 2 (BCL-2) inhibitors. A mutation in the BCL-2 gene allows cancer cells to evade “programmed cell death.” This mutation has been linked to chemotherapy resistance and poor outcomes. One drug under study is venetoclax (Venclexta®), a BCL-2 inhibitor that binds to the cancer cell and leads to apoptosis (cell death).
    • IDH1 and IDH2 inhibitors. Mutations in IDH1 and IDH2 genes cause cells to remain immature and multiply too quickly. Ivosidenib (AG-120), an IDH1 inhibitor, and enasidenib (AG-221), an IDH2 inhibitor, are being studied.
    • Histone deacetylase (HDAC) inhibitor. Histone deacetylase (HDAC) inhibitors are agents that cause a chemical change that stops cancer cells from dividing. Valproic acid (Depakene®) is an HDAC inhibitor that is being studied.
    • Erythroid maturation agents (EMAs). “Erythropoiesis” is the formation of red blood cells in blood-forming tissue. In some blood disorders, such as myelodysplastic syndromes, there are defects in the later stages of red blood cell maturation. These defects lead to a decrease in the formation of healthy red blood cells and to the development of chronic anemia. Luspatercept (ACE-536) is a fusion protein that targets defects in the process of red blood cell (erythrocyte) maturation by blocking transforming growth factor beta (TGFβ). This drug is known as an “erythroid maturation agent” and is being studied in clinical trials.
    • Cell signaling pathway inhibitor. Rigosertib (Estybon™), a small molecule agent that promotes MDS cell death by blocking important cellular pathways, is being studied in trials.
  • Novel hypomethylating agents (HMAs). New formulations of HMAs are under study in clinical trials for treatment of myelodysplastic syndromes. Guadecitabine (SGI-110), a drug administered subcutaneously, is being studied. The oral formulation of azacitidine (CC-486) and the oral formulation of decitabine (ASTX/27) allow long-term, lower-dose regimens that may enhance therapeutic activity by increasing exposure to the cancer cells.
  • Immunotherapy. This is a type of biological therapy that is designed to either boost or suppress the immune system to help the body fight cancer. It uses substances made either by the body or in a laboratory to improve, target or restore immune system function.
    • Immune checkpoint inhibitors. A vital part of the immune system is its ability to distinguish between healthy cells in the body and those that are foreign or harmful. The immune system depends on multiple checkpoints. “Checkpoints” are molecules on certain immune cells that need to be activated in order to start an immune response. Cancer cells sometimes take advantage of these checkpoints to escape the detection of active immune cells. Nivolumab (Opdivo®) (anti-PD1), ipilimumab (Yervoy®) (antiCTLA-4) and atezolizumab (TecentriQ®) (anti-PDL-1) are all checkpoint inhibitors being studied in clinical trials.
    • Vaccine therapy. Researchers are developing vaccines made from proteinbuilding blocks called “peptides,” that can be customized for individual patients to stimulate a strong immune response against their cancer. The Wilms tumor 1 (WT1) antigen has been shown to be expressed on leukemic cells and to be associated with a poor prognosis both in AML andmyelodysplastic syndromes. Researchers continue to evaluate the efficacy of an MDS vaccine in treating patients who have high-risk MDS.

Studies to Improve Assessment of Quality of Life for MDS Patients

Due to the chronic nature of myelodysplastic syndromes and the lack of curative options, quality of life is a major focus of treatment decisions. It is important for doctors to be able to measure how a myelodysplastic syndrome and its treatments are affecting a patient’s daily life. Scientists at the Dana-Farber Cancer Institute used input from patients, caregivers, and healthcare providers to create the Quality of Life in Myelodysplasia Scale (QUALMS). Researchers are using QUALMS to determine how different treatments affect patients’ quality of life. Please visit for more information.

Study to Improve Prognosis in Myelodysplastic Syndromes

A team of researchers at the Cleveland Clinic is developing a personalized, machine learning-based prediction model to help doctors to classify patients with MDS into risk categories. Currently, doctors use the International Prognostic Scoring System-Revised (IPSS-R) to assess risk. However, according to recent data, this system may either underestimate or overestimate risk in up to one-third of patients. The Cleveland Clinic prediction model is designed to incorporate individual patient genomic and clinical data using an algorithm to better predict survival probabilities and outcomes. The patients’ data are entered into a Web application that runs the personalized prediction model and provides overall survival and AML transformation probabilities at different time points that are specific to each patient. To further improve the model, researchers are gathering feedback from clinicians and working to incorporate more outcomes, such as quality of life, into the model.

The National Myelodysplastic Syndromes Study

The purpose of the National Myelodysplastic Syndromes Study is to build a resource that scientists can use to learn more about MDS. This resource will enable scientists to conduct research on how to improve the ways in which myelodysplastic syndromes are diagnosed and treated. The study is enrolling patients who have either suspected or newly diagnosed myelodysplastic syndrome, myelodysplastic syndromes/ myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Study participants will donate biological samples (such as bone marrow and blood) and provide medical information, including details of their signs and/or symptoms, how they perceive their quality of life and the treatments they receive. Study participants may be monitored for life. Please visit The National Myelodysplastic Syndromes Study to learn more.





Related Links