Antitumor activity of rapamycin in a Phase I trial for patients with recurrent PTEN-deficient glioblastoma

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Rapamycin has anticancer activity in PTEN-deficient glioblastoma and warrants further clinical study alone or in combination with PI3K pathway inhibitors. The short-term treatment endpoints used in this neoadjuvant trial design identified the importance of monitoring target inhibition and negative feedback to guide future clinical development.

http://www.ClinicalTrials.gov (#NCT00047073).