Molecular therapeutic approaches for pediatric acute myeloid leukemia.

TitleMolecular therapeutic approaches for pediatric acute myeloid leukemia.
Publication TypeJournal Article
Year of Publication2014
AuthorsTasian SK, Pollard JA, Aplenc R
JournalFront Oncol
Volume4
Pagination55
Date Published2014 Mar
ISSN2234-943X
Abstract

Approximately two-thirds of children with acute myeloid leukemia (AML) are cured with intensive multi-agent chemotherapy. However, refractory and relapsed AML remains a significant source of childhood cancer mortality, highlighting the need for new therapies. Further therapy intensification with traditional cytotoxic chemotherapy in pediatric AML is not feasible given the risks of both short-term and long-term organ dysfunction. Substantial emphasis has been placed upon the development of molecularly targeted therapeutic approaches for adults and children with high-risk subtypes of AML with the goal of improving remission induction and minimizing relapse. Several promising agents are currently in clinical testing or late preclinical development for AML, including monoclonal antibodies against leukemia cell surface proteins, kinase inhibitors, proteasome inhibitors, epigenetic agents, and chimeric antigen receptor engineered T cell immunotherapies. Many of these therapies have been specifically tested in children with relapsed/refractory AML in Phase 1 and 2 trials with a smaller number of new agents under Phase 3 evaluation for children with de novo AML. Although successful identification and implementation of new drugs for children with AML remain a formidable challenge, enthusiasm for novel molecular therapeutic approaches is great given the potential for significant clinical benefit for children who do not have other curative options.

DOI10.3389/fonc.2014.00055
Alternate JournalFront Oncol
PubMed ID24672775
PubMed Central IDPMC3957536
Grant ListT32 CA128583 / CA / NCI NIH HHS / United States