Smoothened inhibitor erismodegib combined with nilotinib in patients with chronic myeloid leukemia resistant/intolerant to at least one prior tyrosine kinase inhibitor: a phase 1b study.

Patients completing 12 cycles (dose escalation/dose expansion) had the option to enter the extension phase to continue treatment until disease progression or onset of unacceptable toxicities. The hedgehog (Hh) pathway is activated in BCR-ABL1 + leukemic stem cells (LSCs) and differentiated hematopoietic cells, through a mechanism involving membrane protein smoothened (SMO) upregulation [[1]]. Overall, 11 patients with CML-CP were enrolled and treated (first patient screened: 5 January 2012; last patient last visit: 5 February 2014). [Extracted from the article]