Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults. Each year in the United States, about 19,900 people (usually older than 45 years of age) are diagnosed, and about 10,400 people die from all forms of the disease, according to the American Cancer Society. Unlike other cancers that start in an organ and spread to the bone marrow, AML is known for rapid growth of abnormal white blood cells that gather in the bone marrow, getting in the way of normal blood cell production. The lack of normal blood cells can cause some of the symptoms of AML, including anemia (shortage of red blood cells resulting in tiredness and weakness), neutropenia (shortage of white blood cells that may lead to increased infections), and thrombocytopenia (shortage of platelets in the blood that may lead to excessive bleeding). Current treatment options for AML consist of reducing and eliminating cancer cells mainly through chemotherapy, radiation therapy, and stem cell transplantation.
GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with AML cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. Preclinical research points to the drug’s potential role in moving cancerous cells out of the protective environment of the bone marrow where they hide and escape the effects of chemotherapy. In preclinical studies using animal models of AML, the results of which were presented at meetings of the American Society of Hematology (ASH), GMI-1271 improved survival in combination with chemotherapy, compared to chemotherapy alone. In preclinical studies, GMI-1271 was also associated with a reduction of chemotherapy-induced neutropenia and chemotherapy-induced mucositis.
GMI-1271 has received Fast Track designation from the U.S. Food and Drug Administration (FDA), which may lead to speedier delivery to people living with AML if the treatment is found to be effective. GMI-1271 has not been approved for use by any worldwide health authority.
About the Phase 1/2 Clinical Trial in AML
GMI-1271 has been tested in healthy volunteers and is now being studied in a Phase 1/2 clinical trial in the U.S. and worldwide. The trial is designed to evaluate GMI-1271’s safety, absorption by the body and effect on AML when given with standard chemotherapy. The drug is being given to trial participants throughout their chemotherapy treatment period. Some additional bone marrow and blood samples are being obtained during routine bone marrow assessments, to learn more about drug activity.
The Phase 1 dose escalation portion of the on-going trial has been completed, and the Phase 2 portion is underway. Results from the Phase 1 portion were reported at the annual meeting of the European Hematology Association in June 2016. Additional findings showing high rates of remission and favorable tolerability among study participants were reported during a poster session at the American Society of Hematology Annual Meeting in December 2016.
Clinical Trial Eligibility
The trial is open to both males and females with AML (including secondary AML).
Trial Contact Information
Speak to your doctor to see if this clinical trial might be right for you. For more information on the trial and to find a participating site near you, please visit: https://clinicaltrials.gov/ct2/show/NCT02306291?term=gmi-1271&rank=2
Clinical Trial Resources
To learn more about clinical trials, please visit:
National Cancer Institute: http://www.cancer.gov/about-cancer/treatment/clinical-trials
American Cancer Society: http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/index
Download Acute Myeloid Leukemia Brochure
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