Investigational Therapies
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Researchers are continually testing new treatments for MDS, and many of these studies originate at Memorial Sloan-Kettering. These investigational approaches are offered to eligible patients here through the clinical trial process, which is designed to advance the current standard of care for MDS.
As with all cancer treatment at Memorial Sloan-Kettering, clinical trials involve a team approach: clinicians, scientists, and pathologists work together to care for each patient. We are expert at determining which patients will derive the most benefit from which trial -- whether it is of chemotherapy or immune therapy or a combination of approaches.
In an effort to improve patients' response rates, Memorial Sloan-Kettering researchers are currently studying various drug combinations for the treatment of MDS, including agents newly available for MDS and one soon to be approved by the FDA. Agents under study include the recently approved drug 5-azacytidine (Vidaza™) and decitabine, another investigational agent for the treatment of MDS, which is likely be FDA-approved in early 2006.
A new drug called 5-azacytidine (Vidaza™) was approved by the FDA in 2004 for the treatment of MDS. 5-azacytidine works by delaying the progression of MDS to acute myelogenous leukemia. In clinical trials, 5-azacytidine improved survival rates among MDS patients. Physicians generally prescribe this drug for patients who have significantly low blood counts (cytopenias including neutropenia, thrombocytopenia, and transfusion-dependent anemia).
Lenalidomide (Revlimid®) is one of a group of drugs called immunomodulatory agents, which work by modifying or regulating the functioning of the immune system. Lenalidomide is related to the drug thalidomide but lacks many of the side effects of that drug. In clinical studies, many MDS patients who had been dependent on red blood cell transfusions no longer needed transfusions when they were treated with lenalidomide. It is especially effective in patients with the specific chromosomal abnormality 5q- syndrome. Two-thirds of patients with this form of MDS responded to treatment. Lenalidomide's mechanism of action is not precisely known. Lenalidomide was approved by the FDA for the transfusion-dependent 5q- patients in 2005.
Decitabine (Dacogen™) is related to the recently approved drug for MDS, 5-azacytidine (Vidaza). Both drugs (hypomethylating agents) appear to promote the maturation, or differentiation, of myeloid cells, and as a result, both drugs prevent or slow the progression of MDS toward leukemia, thereby prolonging the lives of MDS patients.
Another treatment approach, immunosuppression, uses ATG (antithymocyte globulin) to suppress the activity of white cells that, in some cases of MDS, interfere with normal blood cell production. This approach works temporarily in slightly less than half of patients.
For patients whose disease closely resembles leukemia, physicians may use antileukemia chemotherapy.
For up-to-date information about our clinical trials for patients with MDS, please visit our clinical trial database.