Novel therapies and immunotherapeutic approaches to treat childhood leukemia

Document Type

Book Chapter





Publication (Name of Journal)

Frontiers in Clinical Drug Research: Hematology


Haematology/Oncology; Pathology and Laboratory Medicine


Bentham Science


Sharjah, U.A.E


One third of all paediatric cancer is acute leukaemia which includes acute lymphoblastic and acute myeloid leukaemia. Acute lymphoblastic leukaemia accounts for 75% of cases. Multiple genetic factors contribute in predisposing a child to the development of leukaemia. The major scope of research has been to design therapies that improve overall survival in paediatric patients with high risk or relapsed acute leukemias. This is the largest population of children who are refractory to current treatment modalities. The main aim of novel and immunotherapeutic treatment is target-specific therapies with much decreased toxicities compared to those associated with high dose chemotherapeutic agents. Insights into various “driver” mutations and signaling pathways have led to the discovery of novel agents. Immunotherapeutic agents exploit the mechanism of cytotoxic immunity directed against leukaemic cells. Apart from targeted therapies, haematopoietic stem cell transplant has revolutionized the treatment of acute leukaemias. Particularly in high-risk patients, this treatment modality is the only potential curative option available. This review highlights the upcoming agents that are being used in acute paediatric leukaemias. Topics include nucleoside analogues, monoclonal antibodies, CAR T cells, tyrosine kinase inhibitors, epigenetic agents and proteasome inhibitors. The benefit of allogeneic stem cell transplant in high risk or relapsed/refractory patients with acute leukaemia has also been summarized.