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Inhibitors of Caspase 1 for the Treatment of Numerous Autoimmune and Inflammatory Diseases

A nitrile-containing propionic acid moiety as an electrophile for covalent attack by the active site cysteine residue of caspase 1 was investigated. Several cyanopropanate-containing small molecules were synthesized, including one based upon the optimized peptidic scaffold of the prodrug VX-765. A number of these compounds were potent inhibitors of caspase 1 (IC50s d 1nM). Examination of these small molecules versus a caspase panel demonstrated an impressive degree of selectivity for caspase 1 inhibition. A number of these compounds were assessed for their hydrolytic stability and selected ADME properties.

Structure of ML132

Key Investigators

National Center for Advancing Translational Sciences
Matthew B. Boxer, Ph.D.
Min Shen, Ph.D.
Douglas S. Auld, Ph.D.
Craig J. Thomas, Ph.D.

University of California, San Francisco
James A. Wells, Ph.D.

Public Health Impact

Caspase 1 plays an active role in the inflammatory immune response. This probe compound serves as a pivotal starting point to further understand the mechanisms that underlie the immune response in autoimmune and inflammatory diseases, including neurological diseases.

Publication

Boxer MB, Quinn AM, Shen M, et al. A highly potent and selective caspase 1 inhibitor that utilizes a key 3-cyanopropanoic acid moiety. Chem Med Chem, 2010;5:730-738.

Probe Report

A small molecule inhibitor of Caspase 1 Probe Report