Chronic myeloid leukaemia. A paradigm for malignancy or just a strange disease?

ABSTRACT

Chronic myeloid leukaemia [CML], previously a fatal illness, is now readily manageable with oral medication. First described in the 1840s, there was no widely accepted cure until the advent of allogeneic stem cell transplantation in the late 1970s. This treatment was of limited value because of donor availability and toxicity problems. Discovering the Philadelphia chromosome and demonstrating that the BCR-ABL chimaeric gene was responsible for the malignant phenotype opened new avenues. The development of tyrosine kinase inhibitors [TKIs] changed the lives of patients with CML. The treatment has been so successful that compliance is now a problem. Currently under discussion is the possible use of more expensive second generation TKIs for newly diagnosed patients. In spite of the success with TKIs, treatment of common cancers has not been so successful. Is CML therefore a paradigm for malignancy or just a strange disease?