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Druggable signaling pathways in juvenile pilocytic astrocytoma

Chuck Stiles, M.D.

November 2010, Summary

With the generous support of the Childhood Brain Tumor Foundation, the laboratory of Dr. Charles Stiles at Dana-Farber Cancer Institute has developed pre-clinical models for testing drugs that target the PI3K signaling pathway. We have evidence that the PI3K signaling pathway may be mutated in some juvenile pilocytic astrocytomas. We have further used these models to test three separate PI3K antagonists from two different pharmaceutical firms. As a result of our work with these agents, Dr. Patrick Wen at Dana-Farber Cancer Institute has been approved to conduct a first-in-man clinical trial of a Novartis drug known as BKM120 for patients with recurrent glioma. Although this drug will be tested initially on adult patients, it is important to realize that positive results from an adult trial will pave the way for clinical trials of a new drug for pediatric cancers.  Thus, the grant we received from the Childhood Brain Tumor Foundation has enabled the most positive possible outcome for this work. It is our ultimate hope that your support will culminate in a clinical trial of a promising new drug for JPAs. 

On behalf of Dr. Stiles, we are very pleased to provide you with the attached progress report and a short summary below. Please contact us again any time if you need further details. Thanks again for the CBTF’s wonderful support!

Category: Grant Summaries, Research
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