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Pediatric Cancer
Working towards a 100% cure rate for every child

Pediatric oncology has come a long way since the 1960s when a diagnosis of cancer was an automatic death sentence. Today, approximately 75 percent of all pediatric cancer cases are cured, and in some specific diseases, cure rates are as high as 90 percent. But even in those cases it is the unlucky one in 10 that keeps pediatric oncologists up at night. As Dr. Julia Glade Bender, a Columbia pediatric oncologist says, "We can't be satisfied until we get a 100 percent cure rate for every child.

Just as with adult cancer, pediatric cancers are typically treated with surgery, chemotherapy and radiation therapy. However, pediatric tumors are biologically distinct from adult cancers and include unique diagnoses such as neuroblastoma, a tumor originating in cells destined to be nerves, or Wilms, a tumor of the kidney.

"Pediatric oncologists are victims of our own success," says Dr. Bender. Because these cancers are so often curable and because the numbers of children who get these cancers is relatively small compared to adults - just 14,000 new cases per year - it's much more difficult to conduct clinical trials in children. This is particularly challenging when testing dosing for new drugs in order to figure out how to fit promising new treatments into upfront therapy, with the goal of finding more effective curative regimens with the fewest possible side effects.

Two of Dr. Bender's colleagues, Dr. Jessica Kandel, associate professor of surgery, and Dr. Darrell Yamashiro, Irving Assistant Professor of pediatrics, pathology, and surgery, teamed up in 1998 to form the Pediatric Tumor Biology Laboratory. They decided to test anti-angiogenesis treatments, sometimes called "smart drugs," which cut off blood supply to a tumor, effectively starving it to death.

Working with mouse models of pediatric Wilms tumors, they tested the use of several anti-angiogenesis therapies that block VEGF (vascular endothelial growth factor), a substance that tumors release to promote vessel growth.

Their research quickly showed promising results. After six weeks of treatment with the anti-VEGF antibodies, the researchers found that the Wilms tumors hardly grew at all, only reaching a size about 1 percent to 5 percent of the untreated control tumors, and were much less prone to metastasis.

Additionally, within 24 hours of treatment with VEGF-Trap, the drug began to destroy blood vessels inside the tumors, and after five weeks the tumors and the metastases had shrunk by 80 percent.

Based on these promising laboratory findings with mouse models, Dr. Bender is currently leading the first trial with an anti-angiogenesis therapy in children, which so far has shown promising results. She hopes to launch a multi-center study of the anti-angiogenesis therapy in the summer of 2006.

"We have to keep pulling out all the stops so that we give every child a chance at a life," says Dr. Bender.
© 2005 Columbia University