New clues toward treating pediatric brain tumors harboring epigenetic mutation

In mouse models of H3.3G34R/V glioma, a small molecule inhibitor of STAT3 called WP1066 was shown to suppress tumor growth and significantly improve how long the mice survived.

The drug compound has the important property of being able to cross the blood-brain barrier, which is crucial for the development of treatments for brain cancer, and is currently being tested in clinical trials for glioblastoma in adult patients, Venneti adds.

“Our goal is to move the compound into clinical trials for pediatric patients,” he said.

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The work was supported by several federal grants from the National Institutes of Health and the National Cancer Institute, and by organizations such as Matthew Larson, St. Baldrick’s, Claire McKenna, Alex’s Lemonade Stand, Storm The Heavens, Hyundai Hope on Wheels, Michael Mosier Defeat. DIPG, ChadTough, Robert Connor Dawes/National Brain Tumor Society/Ependymoma Cancer Research Network, Sidney Kimmel, Doris Duke and Sontag Foundations.

Other authors include Chan Chung, Siva Kumar Natarajan, Pooja Panwalkar, Matthew Pun, Amer Ghali, Jill Bayliss, Drew Pratt, Anand Shankar, Visweswaran Ravikumar, Arvind Rao, Marcin Cieslik, Kari Wilder-Romans, Andrew J. Scott, Daniel R. Wahl, Selin Jessa, Claudia L. Kleinman, Nada Jabado, Alan Mackay, Chris Jones, Daniel Martinez, Mariarita Santi, Alexander R. Judkins, Viveka Nand Yadav, Tingting Qin, Timothy N. Phoenix, Carl J. Koschmann, Suzanne J. Baker and Arul M. Chinnaiyan.

Cited article: “Epigenetically defined therapeutic targeting in H3G34R/V high-grade gliomas”, Science Translational Medicine. DOI: 10.1126/scitranslmed.eabf7860

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