Pharmacology and Experimental Therapeutics Section
 The
primary mission of the Pharmacology and Experimental Therapeutics Section
of the Pediatric Oncology Branch is to develop new anticancer drugs for
the treatment of childhood cancers. The Pharmacology and Experimental
Therapeutics Section takes an integrated approach to the development of
new drugs that incorporates laboratory studies in both in vitro and animal
models followed by clinical trials which include pharmacokinetic studies
(studies that determine what happens to the drug after it is administered
to patients) in children with cancer. Current research has focused on:
- Development
of new drugs for childhood cancer which differ in their mechanism of
action from the chemotherapeutic agents currently available
- The
development of new agents to treat brain tumors and cancers which have
spread to the meninges (the covering of the brain and spinal cord)
- The
clinical pharmacology of anticancer drugs commonly used in the treatment
of childhood cancers to determine if they are being utilized optimally
- The
development of more effective and less toxic treatment regimens for
childhood acute lymphoblastic leukemia (ALL), with an emphasis on devising
non-radiation based central nervous system preventive therapy and new
approaches to improve the outcome in children with leukemia.
As part of its work, the Pharmacology
and Experimental Therapeutics Section performs a number of clinical trials
which allow children with cancer, in whom standard therapy has failed, to
receive new drugs under development for cancer patients. These studies are
both Phase 1 and Phase 2 clinical trials. Currently, pediatric Phase
1 trials using the following drugs are under study:
- R115777 ( Farnesyltranstease Inhibitor )
- TLCD99 ( Liposomal Doxorubicin
)
- Arsenic Trioxide
- 06Benzylguanine with
Temozolomide
- XR9576 ( P-Glycoprotein
Inhibitor
- BM5247550 ( Epitholone
)
Phase 2 trials underway
include all-trans-retinoic acid/interferon.
For children with meningeal
leukemia or other cancers which have spread to the meninges, Phase
1 & 2 trials include intrathecal
- Mafosfamide
- Topotecan
- Gemcitabine
Frank
M. Balis, M.D., Pharmacology & Experimental Therapeutics Section
Brigitte
Widemann, M.D. Pharmacology & Experimental Therapeutica Section
Last Updated:
July 26, 2006
NCI
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