Vismodegib is a drug approved for a form of skin cancer called basal cell carcinoma. It works by targeting a protein pathway called the “hedgehog” pathway. Vismodegib inhibits SMO, a protein which fuels the growth of some cancers. It is a capsule that is taken orally (by mouth).
In this study, researchers are assessing the safety and effectiveness of vismodegib in patients with acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) that have persisted or returned despite other treatment. Laboratory studies have shown that vismodegib may kill leukemia cells by blocking the hedgehog pathway, which is important for leukemia growth.