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.
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Current MDS Research and Clinical Trials
Below are some of the types of MDS research and trials under way:
- Several combinations of FDA-approved drugs, such as azacitidine and decitabine as well as AML-type chemotherapy agents are being studied in several clinical trials. Each drug works in different ways to kill cancer cells. When drugs are used together, they may kill more MDS cells or they may be as effective as standard MDS therapies, but have less toxic side effects.
- Rigosertib (Estybon™), a drug that promotes MDS cell death, is being researched in current trials for the treatment of intermediate –1, intermediate –2 or high-risk patients as a single agent. It is also being studied in patients whose MDS has stopped responding to azacitidine or decitabine.
- Luspatercept (ACE-536) is a type of TFG (transforming growth factor)-beta inhibitor. This drug is showing promising results in current trials for very lowto intermediate-risk MDS, in patients with the MDS-RS subtype and/or the SF3B1 mutation and who require frequent transfusions.
- Valproic acid (Depakene®), a histone deacetylase (HDAC) inhibitor, is being studied in combination with decitabine (Dacogen®) in the treatment of patients with high-risk MDS.
- Two current clinical trials are investigating the use of programmed death ligand 1 (PD-L1) inhibitors in MDS. Pembrolizumab (Keytruda®), a PD-L1 inhibitor approved for melanoma, is undergoing a trial as a single agent against MDS and certain types of lymphoma. Another PD-L1 inhibitor, atezolizumab (Tecentriq®), is being tested with and without azacitidine in an ongoing trial.
- The CTLA-4 inhibitor ipilimumab (Yervoy®), approved in melanoma, is being studied for treatment of relapsed and refractory MDS before or after stem cell transplantation
Clinical trials are underway to see if an MDS vaccine is effective in treating patients who have higher-risk MDS. The vaccine is made from protein-building blocks called “peptides,” which may help the body mount an effective immune response to MDS cells.