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Ivosidenib for acute myeloid leukaemia with IDH1 mutation


Cancer and Palliative Care

September 2018

Ivosidenib is an oral treatment in clinical development for people with acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation. AML is a type of cancer that causes the bone marrow to produce excess immature white blood cells. Most patients with AML are treated with standard chemotherapy. AML that is non-responsive to treatment is called refractory while that which returns after response to initial treatment is called relapsed. AML in general, and particularly relapsed or refractory disease, is associated with a poor prognosis. In blood cancers such as AML, certain genetic mutations such IDH1 can occur.
Ivosidenib belongs to a new class of therapies that works by inhibiting the mutated IDH1 enzyme, which in turn reduces the level of d-2-hydroxyglutarate (2-HG), an oncometabolite which impairs myeloid differentiation, increases proliferation of myeloblasts, and blocks cellular differentiation. There are currently no approved treatment options in the EU/UK for those who have relapsed or refractory AML with an IDH1 mutation. If licensed, ivosidenib could be an effective precision medicine for this patient group.

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