Journal article

Preclinical Activity and Pharmacokinetic/Pharmacodynamic Relationship for a Series of Novel Benzenesulfonamide Perforin Inhibitors

KH Gartlan, JK Jaiswal, MR Bull, H Akhlaghi, VR Sutton, KA Alexander, K Chang, GR Hill, CK Miller, PD O'Connor, J Jose, JA Trapani, SA Charman, JA Spicer, SMF Jamieson

ACS Pharmacology and Translational Science | Published : 2022

Abstract

Perforin is a key effector of lymphocyte-mediated cell death pathways and contributes to transplant rejection of immunologically mismatched grafts. We have developed a novel series of benzenesulfonamide (BZS) inhibitors of perforin that can mitigate graft rejection during allogeneic bone marrow/stem cell transplantation. Eight such perforin inhibitors were tested for their murine pharmacokinetics, plasma protein binding, and their ability to block perforin-mediated lysis in vitro and to block the rejection of major histocompatibility complex (MHC)-mismatched mouse bone marrow cells. All compounds showed >99% binding to plasma proteins and demonstrated perforin inhibitory activity in vitro an..

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University of Melbourne Researchers