NUI Galway treats first patient in the world as part of clinical trial for promising new treatment in acute leukemia

May 20 2015 Posted: 16:46 IST

NUI Galway has announced that a patient attending University Hospital Galway is the first patient worldwide to start treatment in a clinical study designed to evaluate the safety and efficacy of an investigational drug candidate for sufferers of acute myeloid leukemia. The announcement was made today and co-incided with International Clinical Trials Day.

Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults, and it is estimated that there were over 18,000 new cases and over 10,000 deaths from the disease in 2014 world-wide.

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 and as a result, impede normal blood cell production. While leukemic cells move into the blood, the lack of normal blood cells can cause some of the symptoms of AML such as anaemia and increased risk of infections or excessive bleeding.

Current treatment options for AML are chemotherapy and stem cell transplantation, both of which can destroy cancer cells but do not reduce the related side effects. The investigational drug candidate in the Phase 1/2 clinical study in Galway is being developed by US based company GlycoMimetics, who are exploring the clinical use of the drug candidate in blood cancers. The company announced last week that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to GMI-1271 for treatment of AML.

Professor Michael O’Dwyer, NUI Galway, Principal Investigator on the study, stated that: “Based on preclinical data and on a favourable safety profile in healthy volunteers, we believe that GMI-1271 has the potential to be an important new therapy for people with certain blood cancers. It seems that the therapeutic agent in this potential treatment can reverse resistance to chemotherapy which is caused by AML cells’ ability to bind to a receptor called E-selectin in the bone marrow.”

NUI Galway’s key strategic research priorities include Cancer Research, Regenerative Medicine and Medical Devices. The University is due to open a new facility for patient-centered clinical research and first-in-man clinical trials, as well as translational research, in the coming months.


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