Preclinical models and early phase clinical trials demonstrate that PI3Kα-specific inhibitors exhibit anti-tumor efficacy in cancers with frequent PIK3CA oncogenic mutations, such as lung and colon cancers, but also in hematological cancers such as juvenile myelomonocytic leukemia, acute myeloid leukemia (AML) or chronic lymphocytic leukemia (CLL) [4]. Here, PIK3CA is linked to acute myeloid leukemia.