ABL1 and chronic myelogenous leukemia, BCR-ABL1 positive: This is the first study to demonstrate that a SMO inhibitor suitable for clinical development has efficacy against CP-CML LSC both in vitro and in vivo, and provided preclinical support for clinical trials of LDE225 (Sonidegib) in combination with nilotinib in CP-CML patients who have failed prior therapy with other BCR-ABL tyrosine-kinase inhibitors (NCT01456676).