ONCOLOGY. Vol. 21 No. 7

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Lessons Learned

Pediatric Cancers in the New Millennium: Dramatic Progress, New Challenges

LISA M. McGREGOR, MD, PhD
Assistant Member, Department of Oncology
St. Jude Children' Research Hospital
Assistant Professor, Department of Pediatrics
University of Tennessee Health Science Center College of Medicine

MONIKA L. METZGER, MD
Assistant Member, Department of Oncology
St. Jude Children' Research Hospital
Assistant Professor, Department of Pediatrics
University of Tennessee Health Science Center College of Medicine

ROBERT SANDERS, MD
Assistant Member, Department of Oncology
St. Jude Children' Research Hospital
Assistant Professor, Department of Pediatrics
University of Tennessee Health Science Center College of Medicine

VICTOR M. SANTANA, MD
Member, Department of Oncology
St. Jude Children' Research Hospital
Professor, Department of Pediatrics
University of Tennessee Health Science Center College of Medicine
Memphis, Tennessee

| June 1, 2007

Financial Disclosure: The authors have no significant financial interest or other relationship with the manufacturers of any products or providers of any service mentioned in this article.

There continue to be more challenges than successes in the treatment of pediatric brain tumors. Long-term complications of treatment remain a concern even when tumors are highly curable. Several tumor types, including high-grade and diffuse pontine gliomas, are remarkably resistant to therapy. These and the recently defined atypical teratoid/rhabdoid tumor have a dismal prognosis despite decades of clinical trials.[72,73] For these patients and for those with recurrent medulloblastoma, progress will depend on the elucidation of tumor biology.

To that end, clinical trials are beginning to routinely incorporate biologic studies. To facilitate such studies, neurosurgeons should be encouraged to save frozen tissue from all pediatric brain tumor resections. As tumor-specific molecular abnormalities are identified, new drugs are being developed to target them. Many such agents are now in early pediatric clinical trials in cooperative groups such as COG and the Pediatric Brain Tumor Consortium (PBTC). The effective use of these agents should make it possible to cure a larger proportion of children with brain tumors in the future.

Summary

Fifty years ago, childhood cancer was almost uniformly fatal. Today, most patients survive if treated appropriately. Further progress will require greater coordinated efforts to develop less toxic therapies for the more curable pediatric cancers and novel approaches for patients at greatest risk. There is also a need to establish and support evidence-based national pediatric oncology standards of care, to develop new incentives for drug development specific to childhood cancer, to increase support for translational research, to develop comprehensive management strategies for survivors, and to develop and finance national initiatives to improve the outcome of adolescents and young adults with cancer. Because many remaining research questions will require the study of large populations and access to high-quality biologic specimens, more efficient and productive international collaborations are needed.

Whereas many early clinical trials were empirical, our growing understanding of tumorigenesis, cellular signal transduction, and tumor survival factors is rapidly providing new clues for the development of targeted agents. In addition, new diagnostic and imaging tools promise more robust measurement of disease burden and response to verify the benefits of therapy. The success we have achieved to date is gratifying. We should not be discouraged by our failures but galvanized by the remaining challenges.

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Expert Perspectives on this case report

Curing Pediatric Cancers: A Success Story Reconsidered

This Article Reviewed By
ROBERT J. ARCECI, MD, PhD

The authors would like to thank Sharon Naron for expert editorial review, Catherine Billups for technical support in Figure 1, Dr. William Meyer for providing Figure 4, and Dr. Sheri Spunt for her suggestions.

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TOPIC INDEX

Cancer Types
Breast Breast (HER2+) Breast (Triple-Negative) CML Colorectal Gastrointestinal GIST Genitourinary Gynecologic Head & Neck	Hematology Kidney (Renal Cell) Leukemia Lung Lymphoma Melanoma Multiple Myeloma Ovarian Prostate Sarcoma
Supportive Care	More Topics
Bone Metastases End-of-Life Care Palliative Care	Ethics in Oncology Practice Management Practice & Policy

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