Reversal of Cancer Gene Expression Identifies Repurposed Drugs for Diffuse Intrinsic Pontine Glioma

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Guisheng Zhao, Patrick Newbury, Yukitomo Ishi, Eugene Chekalin, Billy Zeng, Benjamin S. Glicksberg, Anita Wen, Shreya Paithankar, Takahiro Sasaki, Amreena Suri, Javad Nazarian, Michael E. Pacold, Daniel J. Brat, Theodore Nicolaides, Bin Chen, Rintaro Hashizume


Diffuse intrinsic pontine glioma (DIPG) is an aggressive tumor that is very hard to treat with chemotherapy and radiation and impossible to remove with surgery. To increase positive outcomes for patients, researchers worked to identify new potential therapies through drug screening. Once identified, the team tested those drugs against DIPG cell lines. As a result of this work, researchers have identified clinically-available drugs that showed potency against the DIPG cell lines including Triptolide and mycophenolate mofetil (MMF) The approach used in this study is novel and demonstrates an ability to decrease the cost and time normally required in drug discovery. Twenty-eight raw DIPG RNAseq samples provided by CBTN made this work possible.