Novel Therapies and Immunotherapeutic Approaches to Treat Childhood Leukemia
Pp. 184-219 (36)
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
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.
ALL, AML, Bispecific antibody, CAR T cells, Childhood leukaemia,
CLL, CML, Developing country, Epigenetic agents, Gemtuzumab ozogamicin,
Leukaemia biology, Monoclonal antibodies, MLL leukemia, Mutations,
Nucleoside analogues, Pakistan, Proteasome inhibitor, Stem cell transplant,
Tyrosine kinase inhibitor, WHO.
Department of Pathology & Laboratory Medicine/Oncology, Aga Khan University, Karachi, Pakistan.