DOI: 10.1002/cbic.200900698
Haloacetamidine-Based Inactivators of Protein Arginine Deiminase 4
(PAD4): Evidence that General Acid Catalysis Promotes Efficient Inactivation
Bryan Knuckley,[a] Corey P. Causey,[a] Perry J. Pellechia,[a] Paul F. Cook,[b] and Paul R. Thompson*[a]
Dysregulated protein arginine deiminase (PAD) activity, particu-
larly PAD4, has been suggested to play a role in the onset and
progression of numerous human diseases, including rheuma-
toid arthritis (RA). Given the potential role of PAD4 in RA, we
set out to develop inhibitors/inactivators that could be used to
modulate PAD activity and disease progression. This effort led
to the discovery of two mechanism-based inactivators, denot-
ed F- and Cl-amidine, that inactivate PAD4 by the covalent
modification of an active-site cysteine that is critical for cataly-
sis. To gain further insights into the mechanism of inactivation
by these compounds, the effect of pH on the rates of inactiva-
tion was determined. These results, combined with the results
of solvent isotope effect and proton inventory studies, strongly
suggest that the inactivation of PAD4 by F- and Cl-amidine
proceeds by a multistep mechanism that involves the protona-
tion and stabilization of the tetrahedral intermediate formed
upon nucleophilic attack by the active-site cysteine, that is,
Cys645. Stabilization of this intermediate would help to drive
the halide-displacement reaction, which results in the forma-
tion of a three-membered sulfonium ring that ultimately col-
lapses to form the inactivated enzyme. This finding—that pro-
tonation of the tetrahedral intermediate is important for
enzyme inactivation—also suggests that, during catalysis, pro-
tonation of the analogous intermediate is required for efficient
substrate turnover.
In nature, a myriad of post-translational modifications are
found in proteins. These modifications, and the requisite modi-
fying enzymes, can have far-reaching effects on living systems.
Within the family of protein-modifying enzymes are the pro-
tein arginine deiminases (PADs). These enzymes catalyze the
hydrolysis of arginine residues to form citrulline.[1–3] Much
effort from our laboratory has been focused on gaining insight
into the mechanism of the PADs, and, in particular, PAD4.[3–5]
Our interest in PAD4, and the PADs in general, was piqued as
evidence emerged that linked dysregulated PAD activity to the
increased incidence and severity of rheumatoid arthritis
(RA).[2,3,6] This disease, which afflicts nearly 1% of the popula-
tion, is an autoimmune disorder that appears to be triggered,
at least in part, in response to aberrant citrullination, a result
of dysregulated PAD activity. Based on this apparent causal
relationship, we set out to develop inhibitors/inactivators that
could be used to modulate PAD activity and disease progres-
sion. This effort led to the discovery of two mechanism-based
inactivators, denoted F- and Cl-amidine, which are the most
potent PAD inhibitors described to date.[7,8] At the same time,
Fast and colleagues reported that 2-chloroacetamidine, that is,
the warhead in Cl-amidine, also inactivates PAD4 and other
members of the guanidinium-modifying family of enzymes.[9]
Much work has been done to characterize the mechanism of
inactivation, including dialysis experiments to verify irreversible
inhibition. Additionally, analysis of the kinetics of inactivation
demonstrated that F- and Cl-amidine possess kinact/KI values of
3000 and 13000mꢀ1 minꢀ1, respectively.[7,8] Subsequent crystal-
lographic data confirmed that inactivation was due to the co-
valent modification of an active-site cysteine (Cys645) that is
critical for catalysis—this residue promotes arginine deimina-
tion by covalent catalysis according to a mechanism that is
analogous to that of the Cys proteases.[5] While this finding
conclusively demonstrated the mode of inactivation–alkylation
of the thiolate, the precise mechanism of inactivation has yet
to be established.
Although unclear, the mechanism of inactivation presumably
proceeds through one of at least two routes: direct displace-
ment of the halogen through an SN2 mechanism or initial
attack on the iminium carbon, followed by displacement of
the halide to form a sulfonium ring, and ending with concomi-
tant re-formation of the imine and opening of the sulfonium
ring (Scheme 1). While the former possibility is the more intui-
tive, the latter is analogous to the mechanism by which the
fluoromethylketones inactivate the cysteine proteases,[10–12] and
therefore warranted further investigation.
[a] B. Knuckley, C. P. Causey, P. J. Pellechia, Prof. P. R. Thompson
Department of Chemistry and Biochemistry, University of South Carolina
631 Sumter Street, Columbia, SC 29208 (USA)
We began our investigations by examining the influence of
pH on kinact/KI, that is, the second-order rate constant of
enzyme inactivation. These studies were pursued because pH
rate profiles can suggest the identity of catalytically important
functional groups, in the inactivator or free enzyme, up to and
including the first irreversible step of the reaction. For these
studies, kinact/KI values were determined for both Cl- and F-ami-
[b] Prof. P. F. Cook
Department of Chemistry and Biochemistry, University of Oklahoma
620 Parrington Oval, Norman, OK 73019 (USA)
Supporting information for this article is available on the WWW under
http://dx.doi.org/10.1002/cbic.200900698.
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