[Chronic myeloid leukemia – a model disease for targeted therapy].

Chronic myeloid leukemia - a model disease for targeted therapy Chronic myeloid leukemia (CML) pioneered as the first human malignancy linked to a specific cytogenetic aberration (the Philadelphia chromosome), which led the way to specific targeted therapies with imatinib (Glivec) and later tyrosine kinase inhibitors (TKI). Continuous TKI administration, blocking the oncogenic fusion protein Bcr-Abl, has revolutionized the outcome of CML, transforming an almost uniformly deadly disease into a chronic disorder with a near to normal life expectancy for many patients. There are now indications that, in a portion of patients achieving deep molecular responses, TKI treatment can be stopped without signs of relapse, indicating that these drugs may indeed induce cure. This is of particular importance since adverse events related to long-term TKI therapy, compromising quality of life, are now being increasingly recognized. With the recent introduction of generics the price of imatinib has dropped by more than 95% in Sweden, making an already cost effective treatment even more attractive.