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:
Treatment combinations of FDA-approved drugs, such as Vidaza® or Dacogen®, and AML-type chemotherapy 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.
Vidaza is also being studied as maintenance therapy for MDS patients who achieve a complete or partial remission after intensive chemotherapy. The purpose of the study is to see if Vidaza maintenance improves the duration of patient response.
The following list includes examples of drug combinations and single-agent drugs under study:
- Clofarabine (Clolar®) is a drug approved to treat childhood relapsed or refractory acute lymphoblastic leukemia (ALL). It is being studied when given in combination with AML-type chemotherapy.
- Rigosertib (Estybon™), a drug that increases MDS cell death, is being studied with intermediate-1–, intermediate-2– or high-risk patients as a single agent. It is also being studied in phase 3 trials with early data suggesting effectiveness in patients whose MDS has stopped responding to Vidaza or Dacogen.
- Ezatiostat (Telintra®), a glutathione S-transferase P1-1 inhibitor that activates the Jun-kinase pathway is being studied in lower-risk, transfusion-dependent patients as a single agent.
- Valproic acid (Depakene®), a histone deacetylase (HDAC) inhibitor, is being studied in combination with decitabine (Dacogen).
- Vorinostat (Zolinza®), a histone deacetylase (HDAC) inhibitor, is being studied in combination with azacitidine (Vidaza).
- Azacitidine (Vidaza), in combination with lenalidomide (Revlimid®), is being studied for higher-risk or transfusion-dependent patients.
Clinical trials are under way to see if an MDS vaccine is effective in treating patients who are aged 18 years and older and have lower-risk MDS. The vaccine is made from protein-building blocks, called “peptides,” that may help the body mount an effective immune response to MDS cells.
Vaccine therapy and donor lymphocyte infusions are also being studied as treatments for MDS (and other blood cancer) patients who develop progressive or relapsed disease following allogeneic stem cell transplantation.
Reduced-Intensity Stem Cell Transplantation
Patients being conditioned for a nonmyeloablative transplant receive lower doses of chemotherapy drugs and/or radiation. Immunosuppressive drugs are used to prevent rejection of the graft, and the engraftment of donor immune cells may allow these cells to attack the disease (graft-versus-cancer effect). Studies are researching the use of this type of transplant in older adults who have relapsed and/or refractory disease, as well as the use of unrelated umbilical cord blood and haploidentical (“half-matched”) donors.