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Molecularly Targeted Therapy for Pediatric Brain
Tumors
Warren KE
National Cancer Institute, Neuro-Oncology Branch, Bloch Bldg 82, Rm 224,
9030 Old Georgetown Road, 20892-8200, Bethesda, Maryland, USA,
warrenk@mail.nih.gov.
Treatment of pediatric brain tumors remains a challenge because of the
toxicity associated with conventional treatment and the relative resistance
of tumors at the time of recurrence.
The traditional approach of
administering cytotoxic agents at the maximum tolerated dose is being
supplanted by the development of molecularly targeted agents aimed at
critical cellular changes that are responsible for the growth and spread of
cancer cells.
These agents theoretically should be more specific for tumor
cells and less toxic to normal cells.
While the idea of targeted therapy has
generated much excitement in the oncology community, the degree of benefit
to patients with central nervous system (CNS) tumors remains unclear.
Numerous challenges remain in the development of these agents, including
identification of meaningful targets, delivery of agents in sufficient
quantity at the target site, and determination of any biologic response to
these agents.
This article discusses the rationale behind several of these
agents and their use in pediatric patients with brain tumors.
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