Technique could speed new medulloblastoma drugs

Investigators at St. Jude Children's Research Hospital have developed a strategy to speed future development of more effective and less toxic treatments for medulloblastoma, a type of brain cancer. Medulloblastomas arise in the back of the brain and account for about 20 percent of childhood brain tumors.

The new technique could also be used to identify specific pathways in other types of cancer that might be vulnerable to novel therapies, and therefore speed development of so-called molecular-targeted therapies for a wide variety of cancers. Molecular-targeted therapies work by blocking individual molecules that are key triggers of disease.

This St. Jude study is important because the aggressive combination of surgery, radiation and chemotherapy used to treat medulloblastoma fails to cure many patients, according to researchers. Therefore, there is a great need to identify alternative therapies, such as novel drugs that block signaling pathways that are abnormally activated. Such treatments could not only save lives but also eliminate the severe side effects caused by current therapies, according to Richard Gilbertson, M.D., Ph.D., an associate member of the Department of Developmental Neurobiology at St. Jude and director of the Molecular Clinical Trials Core. Gilbertson, co-director of the St. Jude Neurobiology and Brain Tumor Program, is senior author of a paper on this work that appears in the April issue of Journal of Clinical Oncology.

The technique is designed to rapidly match a specific novel drug treatment to those children most likely to respond to it. This approach would avoid trial-and-error therapy that fails in patients who are not ideal candidates for a specific treatment, researchers said. It would also reduce the chance that otherwise effective drugs would be abandoned because they failed in such patients during clinical trials.

The key to the new St. Jude strategy is the ability to determine in individual children which bioch

Contact: Bonnie Kourvelas
St. Jude Children's Research Hospital

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