Ivosidenib is an oral targeted therapy used to treat certain types of acute myeloid leukemia (AML) and other cancers with a specific genetic mutation.
Key target:
- Mutant IDH1 (isocitrate dehydrogenase 1)
Mechanism of action:
In some cancers, mutated IDH1 produces an abnormal metabolite called 2-hydroxyglutarate (2-HG), which blocks normal cell differentiation. Ivosidenib inhibits the mutant IDH1 enzyme, reducing 2-HG levels and allowing leukemia cells to mature into normal blood cells.
Clinical uses:
- AML with IDH1 mutation, especially in:
- Relapsed/refractory AML
- Newly diagnosed AML in adults who are not eligible for intensive chemotherapy
- Also explored in cholangiocarcinoma and other solid tumors with IDH1 mutations
Common side effects:
- Fatigue
- Nausea
- QT prolongation (heart rhythm issue)
- Differentiation syndrome (a serious but treatable complication)
Summary:
Ivosidenib is a precision medicine that targets IDH1-mutant AML, helping cancer cells resume normal maturation and reducing disease progression.