Nilotinib can inhibit the tyrosine kinase activity of the BCR-ABL protein; CML is caused by the BCR-ABL oncogene. Nilotinib can overcome resistance by inhibiting BCR-ABL with higher affinity than imatinib by binding to the ATP-binding site of the BCR-ABL protein.
Nilotinib showed potential use for myeloproliferative diseases like; chronic myelomonocytic leukemia, and hyper eosinophilic syndrome by representing ability to inhibit TEL-platelet-derived growth factor receptor-beta (TEL-PDGFRbeta), which causes chronic myelomonocytic leukemia, and FIP1-like-1-PDGFRalpha, which causes hyper eosinophilic syndrome. It can also inhibit the c-Kit receptor kinase, like the D816V-mutated variant of KIT, at pharmacologically achievable concentrations, showing potential use in the treatment of mastocytosis, and gastrointestinal stromal tumors.
It is indicated for the treatment of: