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Drug combo shows promise against childhood nerve tumor

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Published: April 29, 2009

A two-drug combination that includes a medicine often used to treated severe acne "significantly" slows the growth of a form of cancer that strikes infants children, early test results suggest.

During last week's annual meeting of the American Society of Pediatric Hematology/Oncology, in San Diego, researchers from the Children's Cancer Hospital at the University of Texas M. D. Anderson Cancer Center, reported that the combination of vandetanib and 13-cis-retinoic acid reduced neuroblastoma tumors by 86 percent in a preclinical test trial.

Vandetanib, is a multikinase inhibitor, a novel class of drug that has shown and ability to suppress the growth of various forms of cancer. The other half of the comb, 13-cis-retinoic acid, or CRA, is probably better known to people with stubborn acne by one of its brand names, Accutane.

"By itself, vandetanib inhibited tumor growth by two-thirds and decreased blood vessel formation around neuroblastoma tumors in mice," says Dr. Peter Zage, a neuroblastoma specialist at the Children's Cancer Hospital, in Houston. "When combined with CRA, the impact was even greater on tumor growth."

Zage, an assistant professor of pediatrics, received this year's Young Investigator Award from the American Society of Pediatric Hematology/Oncology. He is a 1999 graduate of New York's Columbia University College of Physicians and Surgeons.

Neuroblastoma is a malignancy that springs from the tissue of nerves that carry message from the brain to the body and control mainly involuntary functions such as digestion and heartbeat. It is the most common from of cancer infants and the most common form of solid tumor outside the brain in children.

Although doctors use CRA to treat neuroblastoma it has shown no ability to halt the on its own.

Researchers believe vandetanib exerts its anti-cancer powers in at least two ways:

•By blocking a family of endothelial growth factors and receptors that promote tumor growth.

By inhibiting the RET oncogene, which can signal neuroendocrine cells in the autonomic nervous system to develop into neuroblastoma cells.

Currently, there is a Phase I clinical trial open for children with multiple-relapsed neuroblastoma to further study the new therapy combination. This trial is the first in the world to test vandetanib in children.

Zage says in addition to the pediatric patients enrolled in the Phase I trial, adult patients with lung cancer have received vandetanib and have tolerated the novel drug well.

According to the American Cancer Society, approximately 650 children, mainly under the age of 5, are diagnosed with neuroblastoma in the United States each year. About two-thirds of these children are diagnosed after the cancer has spread to other parts of the body.

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