Oncotarget

Research Papers:

Structural basis for small molecule targeting of the programmed death ligand 1 (PD-L1)

Krzysztof M. Zak, Przemyslaw Grudnik, Katarzyna Guzik, Bartosz J. Zieba, Bogdan Musielak, Alexander Dömling, Grzegorz Dubin and Tad A. Holak _

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Oncotarget. 2016; 7:30323-30335. https://doi.org/10.18632/oncotarget.8730

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Abstract

Krzysztof M. Zak1,2, Przemyslaw Grudnik2, Katarzyna Guzik3, Bartosz J. Zieba1,3, Bogdan Musielak3, Alexander Dömling4, Grzegorz Dubin1,2 and Tad A. Holak1,3,5

1 Malopolska Centre of Biotechnology, Jagiellonian University, Gronostajowa, Krakow, Poland

2 Faculty of Biochemistry, Biophysics and Biotechnology, Jagiellonian University, Gronostajowa, Krakow, Poland

3 Department of Organic Chemistry, Faculty of Chemistry, Jagiellonian University, Ingardena, Krakow, Poland

4 Department for Drug Design, University of Groningen, A. Deusinglaan , Groningen, The Netherlands

5 Max Planck Institute for Biochemistry, Am Klopferspitz, Martinsried, Germany

Correspondence to:

Grzegorz Dubin, email:

Tad A. Holak, email:

Keywords: immunotherapy, checkpoint inhibitor, small molecule, X-ray structure

Received: March 30, 2016 Accepted: March 31, 2016 Published: April 13, 2016

Abstract

Targeting the PD-1/PD-L1 immunologic checkpoint with monoclonal antibodies has provided unprecedented results in cancer treatment in the recent years. Development of chemical inhibitors for this pathway lags the antibody development because of insufficient structural information. The first nonpeptidic chemical inhibitors that target the PD-1/PD-L1 interaction have only been recently disclosed by Bristol-Myers Squibb. Here, we show that these small-molecule compounds bind directly to PD-L1 and that they potently block PD-1 binding. Structural studies reveal a dimeric protein complex with a single small molecule which stabilizes the dimer thus occluding the PD-1 interaction surface of PD-L1s. The small-molecule interaction “hot spots” on PD-L1 surfaces suggest approaches for the PD-1/PD-L1 antagonist drug discovery.


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