Our Approach

Publications

We are proud to be collaborating with world-leading academic institutions on advancing treatment options for patients with brain tumors. Notable research that either incorporates testing of our molecules or demonstrates important potential applications of DHODH inhibition in neuro-oncology can be found below:

April 17, 2026

KDM6 Enzymes are the Mechanistic Targets of Mutant IDH that Dictate Replication Stress Sensitivity

Alexander C-Y Tsai, Mathew D Lin, Vinesh T Puliyappadamba, Dorothy M Junginger, Victoria G Donovan, Eleanor G Kaplan, Laura M Drepanos, Hiroaki Wakimoto, Daniel P Cahill, Julie A Losman, Kent W Mouw, Kalil G Abdullah, John G Doench, Duane Nash, Daniel Vitt, Christian Gege, Hella Kohlhof, Samuel K McBrayer, William G Kaelin Jr., Diana D Shi

doi: https://doi.org/10.64898/2026.04.14.715350

August 18, 2022

De novo pyrimidine synthesis is a targetable vulnerability in IDH mutant glioma 

Diana D Shi, Milan R Savani, Michael M Levitt, Adam C Wang, Jennifer E Endress, Cylaina E Bird, Joseph Buehler, Sylwia A Stopka, Michael S Regan, Yu-Fen Lin, Vinesh T Puliyappadamba, Wenhua Gao, Januka Khanal, Laura Evans, Joyce H Lee, Lei Guo, Yi Xiao, Min Xu, Bofu Huang, Rebecca B Jennings, Dennis M Bonal, Misty S Martin-Sandoval, Tammie Dang, Lauren C Gattie, Amy B Cameron, Sungwoo Lee, John M Asara, Harley I Kornblum, Tak W Mak, Ryan E Looper, Quang-De Nguyen, Sabina Signoretti, Stefan Gradl, Andreas Sutter, Michael Jeffers, Andreas Janzer, Mark A Lehrman, Lauren G Zacharias, Thomas P Mathews, Julie-Aurore Losman, Timothy E Richardson, Daniel P Cahill, Ralph J DeBerardinis, Keith L Ligon, Lin Xu, Peter Ly, Nathalie Y R Agar, Kalil G Abdullah, Isaac S Harris, William G Kaelin Jr, Samuel K McBrayer

doi: https://doi.org/10.1016/j.ccell.2022.07.011

 

More Coming Soon

 

Background on DHODH inhibition in Genetic Populations of Brain Tumors

  • DHODH is an independent prognostic marker and potent therapeutic target in neuroblastoma

    Thale Kristin Olsen, Cecilia Dyberg, Bethel Tesfai Embaie, Adele Alchahin, Jelena Milosevic, Jane Ding, Jörg Otte, Conny Tümmler, Ida Hed Myrberg, Ellen M. Westerhout, Jan Koster, Rogier Versteeg, Han-Fei Ding, Per Kogner, John Inge Johnsen, David B. Sykes and Ninib Baryawno

    https://insight.jci.org/articles/view/153836

  • Cancer-selective metabolic vulnerabilities in MYC-amplified medulloblastoma

    William D Gwynne, Yujin Suk, Stefan Custers, Nicholas Mikolajewicz, Jeremy K Chan, Zsolt Zador, Shawn C Chafe, Kui Zhai, Laura Escudero, Cunjie Zhang, Olga Zaslaver, Chirayu Chokshi, Muhammad Vaseem Shaikh, David Bakhshinyan, Ian Burns, Iqra Chaudhry, Omri Nachmani, Daniel Mobilio, William T Maich, Patricia Mero, Kevin R Brown, Andrew T Quaile, Chitra Venugopal, Jason Moffat, J Rafael Montenegro-Burke, Sheila K Singh

    https://pubmed.ncbi.nlm.nih.gov/36368321

  • A druggable addiction to de novo pyrimidine biosynthesis in diffuse midline glioma

    https://www.cell.com/cancer-cell/fulltext/S1535-6108(22)00325-7

  • Revealing de novo pyrimidine synthesis as a key vulnerability in brain tumors

    https://www.cell.com/cancer-cell/fulltext/S1535-6108(22)00520-7

  • Exploiting genetic and environmental vulnerabilities to target DHODH in CNS tumors

    Neuro-Oncology Advances, Volume 7, Issue 1, January-December 2025, vdaf149, doi: https://doi.org/10.1093/noajnl/vdaf149

  • KDM6 Enzymes are the Mechanistic Targets of Mutant IDH that Dictate Replication Stress Sensitivity

    Alexander C-Y Tsai, Mathew D Lin, Vinesh T Puliyappadamba, Dorothy M Junginger, Victoria G Donovan, Eleanor G Kaplan, Laura M Drepanos, Hiroaki Wakimoto, Daniel P Cahill, Julie A Losman, Kent W Mouw, Kalil G Abdullah, John G Doench, Duane Nash, Daniel Vitt, Christian Gege, Hella Kohlhof, Samuel K McBrayer, William G Kaelin Jr., Diana D Shi

    doi: https://doi.org/10.64898/2026.04.14.715350