Acute lymphoblastic leukemia (ALL) is the most frequent cancer in children. The prognosis improved remarkably during the last 50 years due to chemosensitivity of leukemic cells to combined chemotherapy, stratification of treatment according to early treatment response and the introduction of hematopoietic stem cell transplantation (HSCT) into the therapy of high-risk patients.
Despite this improvement, about 10-15% of patients suffer from relapse with only 30-50% chance to be cured, 2-5% of children die due to treatment toxicity and many patients cured by HSCT suffer from late-sequelae. Progress in the development of molecular biology methods allows detection of the abnormal signalling pathways in leukemic cells, enabling the application of targeted therapy and immunotherapy.
These approaches have a potential to improve treatment results due to the decreased incidence of relapses and toxic deaths, and a potential to substitute HSCT in the future.