Development of new therapeutic approaches for leukemia

Ruth Muñoz Risueño


    Ruth Muñoz Risueño


    Institut de Recerca contra la Leucèmia Josep Carreras, Spain


    Identification of immunomodulators, which are already approved for the treatment of immune-related diseases, as potential anti-Acute Myeloid Leukaemia (AML) agents.

    AML is a hematological neoplasia characterized by the rapid expansion of immature myeloid progenitors in the bone marrow. The course of the disease is marked by poor prognosis, frequent relapse, and high disease-related mortality. Survival rates for the majority of patients with AML have not improved, thus new therapeutic approaches are necessary for remission induction and prevention of relapse.

    To reposition already approved drugs for immune disease to treat Acute Myeloid Leukaemia. The anti-AML effect of these drugs has been achieved at a dose within the safety range already approved for other indications.

    Problem to Solve
    Acute Myeloid Leukaemia (AML) is a hematological neoplasia characterized by an abnormal growth and differentiation of immature myeloid cells that accumulate in the bone marrow, hence interfering with the normal production of blood cells. In the past few decades, the main therapeutic options for AML have not changed significantly. Improvement in patient management and optimization of existing drug combinations have accounted for the modest improvement in overall survival rate of patients with AML, with survival rates 5 years after diagnosis still ranking between 30 and 50%. Therefore, new therapeutic options are necessary.

    Pre-clinical studies have shown that these drugs are potent as anti-AML agents. Moreover, the anti-AML effect was achieved within the safety range already approved for other indications.

    Level of Innovation
    The target, a well-known membrane receptor, represents a new therapeutic approach in the field of oncology with a novel mechanism of action. Pre-clinical studies have shown that this receptor is present on all AML patient samples. Thus, already available antagonist for this receptor could be utilized in all patients with AML, as at the present time there is no such overarching treatment of the entire population of patients.