Drug therapy reduces neuroblastoma tumor growth in pre-clinical investigation

April 24, 2009

SAN DIEGO - Researchers from the Children's Cancer Hospital at The University of Texas M. D. Anderson Cancer Center have discovered a new drug combination that significantly hinders tumor growth in neuroblastoma, a childhood cancer. The study was presented today at the 22nd annual meeting of the American Society of Pediatric Hematology/Oncology (ASPHO).

By combining a novel multi-kinase inhibitor, vandetanib, with 13-cis-retinoic acid (CRA), a drug often used for severe acne, researchers from the Children's Cancer Hospital at M. D. Anderson found that the two therapies reduced neuroblastoma tumors by 86 percent in pre-clinical tests.

Peter Zage, M.D., Ph.D., who specializes in neuroblastoma at the Children's Cancer Hospital, received this year's Young Investigator Award from ASPHO and was selected to present his research today in a special platform session.

"By itself, vandetanib inhibited tumor growth by two-thirds and decreased blood vessel formation around neuroblastoma tumors in mice," says Zage. "When combined with CRA, the impact was even greater on tumor growth."

Vandetanib blocks a family of endothelial growth factors and receptors that promote tumor growth as well as the RET oncogene, which can signal neuroendocrine cells to develop into neuroblastoma cells. Although commonly used in treatment for neuroblastoma, CRA by itself does not have a significant impact against the childhood cancer. However, it works in combination therapy to differentiate neuroblastoma cells into mature benign neural 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 five, are diagnosed with neuroblastoma in the United States each year. Close to two-thirds of these children are diagnosed after the cancer has metastasized to other parts of the body. For these patients with high-risk neuroblastoma, long-term survival is less than 40 percent because the tumors are often resistant to traditional chemotherapy.
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About M. D. Anderson

The University of Texas M. D. Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. M. D. Anderson is one of only 41 Comprehensive Cancer Centers designated by the National Cancer Institute. For four of the past six years, M. D. Anderson has ranked No. 1 in cancer care in "America's Best Hospitals," a survey published annually in U.S. News and World Report.

About the Children's Cancer Hospital at M. D. Anderson

The Children's Cancer Hospital at The University of Texas M. D. Anderson Cancer Center has been serving children, adolescents and young adults for more than 50 years. In addition to the groundbreaking research and quality of treatment available to pediatric patients, the Children's Cancer Hospital provides its patients with comprehensive programs that help the children lead more normal lives during and after treatment. For further information, visit the Children's Cancer Hospital Web site at www.mdanderson.org/children.

University of Texas M. D. Anderson Cancer Center

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