How does the POB augment work in the extramural community?
Progress against cancer in children has benefited greatly from well-organized cooperative groups in the extramural community that conduct efficient and large randomized trials. However, future progress hinges upon biologic insight and targeted therapies, both of which begin in a basic science laboratory. The focused basic and clinical science conducted in the POB provides the basis and rationale for new therapies, which, if successful, would ultimately be tested in the extramural community.

In what case would a child, adolescent, or young adult come to clinicians in the POB at the NIH Clinical Center in Bethesda for treatment?
The POB conducts clinical trials targeting a variety of childhood cancers and cancer predisposition syndromes for which outcomes with standard therapies are suboptimal. Any child, adolescent, or young adult who is eligible for such trials may be treated in the POB. In some cases, this represents frontline therapy for a newly diagnosed cancer and in others, the therapies are tested in the setting of recurrent disease. A list of POB protocols can be found at http://pediatrics.cancer.gov/protocol.shtml.

Why is pediatric oncology appropriate for patients who would under other circumstances be considered adults?
Cancers that are most common in children but also afflict adults are often treated by pediatric oncologists familiar with the biology and behavior of those diseases. For example, Ewing sarcoma usually occurs in the second decade of life, but may also occur during early and mid-adulthood and, from a molecular standpoint, is the same disease regardless of the age at which it strikes. Treatment regimens used for adults with this disease are the same as those used for children and therefore many current studies for Ewing sarcoma allow both children and adults to be treated on the same trial.

What are examples of cutting-edge research this year in the POB?
Historically, the Molecular Oncology Section of the POB, under the direction of Dr. Lee Helman, contributed seminal work defining the critical role for IGF-1R signaling in pediatric sarcomas. Monoclonal antibodies targeting this receptor have recently entered phase I clinical trials with encouraging results. There is great optimism in the pediatric sarcoma community that this may represent the first biologically based targeted therapy to show activity in these diseases. In a second example, the Hematologic Diseases Section, under the direction of Dr. Alan Wayne, is working closely with investigators from the Laboratory of Molecular Biology at NCI to develop anti-CD22-based immunotoxins to target acute lymphoblastic leukemia (ALL). A recent phase I study showed clinical activity using this approach in patients with recurrent ALL and plans are underway to improve it with a newer, higher-affinity version of the toxin conjugate and combination with standard chemotherapy, which was synergistic in preclinical models. Recurrent ALL remains the single greatest killer of children with cancer and we are very hopeful that this new biologically based therapy will provide new options for patients.

To learn more about the Pediatric Oncology Branch please visit: http://home.ccr.cancer.gov/oncology/pediatric/.