Taking part in a clinical trial may be a good treatment choice for people with chronic myelomonocytic leukemia (CMML). Clinical trials are under way to help improve treatment, extend survival and improve the quality of life for CMML patients. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society continues to invest funds in CMML research.
Clinical trials can involve new drugs, new combinations of drugs or approved drugs being studied to treat patients in new ways such as new drug doses or new schedules to administer the drugs. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.
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Current CMML Research and Clinical Trials
Below are some examples of clinical trials for new or improved drug therapies currently under study:
ABNL-MARRO STUDY. This study is an initiative from the MDS/MPN International Working Group (IWG) and Vanderbilt University Medical Center. This international collaboration will enroll patients with MDS/MPN overlap syndromes (including CMML) in the United States and several European countries. The study is designed to allow new drugs and therapy combinations to be introduced into the different clinical sites participating. Researchers also seek to study the biology and pathology of the diseases to identify potential markers of prognosis and response.
Monoclonal antibodies. These immunotherapy agents are proteins made in the laboratory that can bind to substances in the body, including cancer cells. They can work alone or can be coupled with a chemotherapy drug or a toxin, or attached to a toxin to kill malignant cells. Examples include monoclonal antibody targeting GM-CSF (lenzilumab™), a cytokine (chemical) to which CMML cells are hypersensitive, and tagraxofusp (Elzonris®) (antibody to CD123, conjugated with diphtheria toxin).
JAK2 Inhibitors. The JAK2 gene makes a receptor (protein) which sends signals into cells that promote cell growth. This protein helps control the number of blood cells made in the bone marrow. Mutated forms of this gene are present in some CMML patients (about 10%); however, the protein is believed to be activated regardless of the presence of the mutation. JAK2 inhibitors target the abnormal protein activation in the cancer cells. Ruxolitinib (Jakafi®) and pacritinib are two examples of JAK2 inhibitors in clinical trials for CMML treatment.
Hypomethylating agents (HMAs). These drugs prevent cells from making DNA, thus inhibiting cell division and making cancer cells more susceptible to death. Decitabine (Dacogen®) and azacitidine (Vidaza®) are two HMAs being studied for use in combination with other agents for CMML treatment. The second-generation HMA guadecitabine is under study for patients with MDS and CMML who have not responded to treatment or have relapsed.
Reduced-intensity allogeneic stem cell transplantation (also known as “nonmyeloablative allogeneic stem cell transplantation”) may prove effective for CMML patients who do not respond to drug therapy but are not considered candidates for allogeneic transplant because of older age or other health risks. Patients being conditioned for a reduced-intensity transplant receive lower doses of chemotherapy and/or radiation than those given to patients before a standard stem cell transplantation. Patients also receive immunosuppressive drugs to prevent rejection of the graft.
The engraftment of donor immune cells may allow these cells to attack the disease (graft-versus-leukemia effect). The theory being tested with a reduced-intensity transplant is that by undergoing less-toxic conditioning prior to the transplant, the body will be better able to withstand the procedure but full donor engraftment still takes place, and the desired graft-versus-leukemia effect still occurs. Relapse is one of the major causes of failure of stem cell transplant for CMML, and clinical trials are currently evaluating combination therapy with azacitidine, lenalidomide, and donor lymphocyte infusions.
- Download or order The Leukemia & Lymphoma Society's free booklet, Chronic Myelomonocytic Leukemia (CMML) and Juvenile Myelomonocytic Leukemia (JMML)