PMID- 25830014
OWN - NLM
STAT- PubMed-not-MEDLINE
DCOM- 20150401
LR  - 20231104
IS  - 2040-6207 (Print)
IS  - 2040-6215 (Electronic)
IS  - 2040-6207 (Linking)
VI  - 6
IP  - 2
DP  - 2015 Apr
TI  - Novel agents for the treatment of childhood acute leukemia.
PG  - 61-79
LID - 10.1177/2040620714565963 [doi]
AB  - Together, acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) 
      make up approximately one-third of all pediatric cancer diagnoses. Despite 
      remarkable improvement in the treatment outcomes of these diseases over the past 
      several decades, the prognosis for certain high-risk groups of leukemia and for 
      relapsed disease remains poor. However, recent insights into different types of 
      'driver' lesions of leukemogenesis, such as the aberrant activation of signaling 
      pathways and various epigenetic modifications, have led to the discovery of novel 
      agents that specifically target the mechanism of transformation. In parallel, 
      emerging approaches in cancer immunotherapy have led to newer therapies that can 
      exploit and harness cytotoxic immunity directed against malignant cells. This 
      review details the rationale and implementation of recent and specifically 
      targeted therapies in acute pediatric leukemia. Topics covered include the 
      inhibition of critical cell signaling pathways [BCR-ABL, FMS-like tyrosine kinase 
      3 (FLT3), mammalian target of rapamycin (mTOR), and Janus-associated kinase 
      (JAK)], proteasome inhibition, inhibition of epigenetic regulators of gene 
      expression [DNA methyltransferase (DNMT) inhibitors, histone deacetylase (HDAC) 
      inhibitors, and disruptor of telomeric signaling-1 (DOT1L) inhibitors], 
      monoclonal antibodies and immunoconjugated toxins, bispecific T-cell engaging 
      (BiTE) antibodies, and chimeric antigen receptor-modified (CAR) T cells.
FAU - Annesley, Colleen E
AU  - Annesley CE
AD  - Oncology and Pediatrics, The Sidney Kimmel Comprehensive Cancer Center, Johns 
      Hopkins University School of Medicine, Baltimore, MD, USA.
FAU - Brown, Patrick
AU  - Brown P
AD  - Oncology and Pediatrics, The Sidney Kimmel Comprehensive Cancer Center, Johns 
      Hopkins University School of Medicine, 1650 Orleans Street, CRB-I 2M46, 
      Baltimore, MD 21231, USA.
LA  - eng
PT  - Journal Article
PT  - Review
PL  - England
TA  - Ther Adv Hematol
JT  - Therapeutic advances in hematology
JID - 101549589
PMC - PMC4365053
OTO - NOTNLM
OT  - ALL
OT  - AML
OT  - CAR T-cells
OT  - EPZ-5676
OT  - FLT3
OT  - blinatumomab
OT  - carfilzomib
OT  - moxetumomab
COIS- Conflict of interest statement: The authors declare that there is no conflict of 
      interest.
EDAT- 2015/04/02 06:00
MHDA- 2015/04/02 06:01
PMCR- 2015/04/01
CRDT- 2015/04/02 06:00
PHST- 2015/04/02 06:00 [entrez]
PHST- 2015/04/02 06:00 [pubmed]
PHST- 2015/04/02 06:01 [medline]
PHST- 2015/04/01 00:00 [pmc-release]
AID - 10.1177_2040620714565963 [pii]
AID - 10.1177/2040620714565963 [doi]
PST - ppublish
SO  - Ther Adv Hematol. 2015 Apr;6(2):61-79. doi: 10.1177/2040620714565963.