The Journal of Experimental Medicine
BRIEF DEFINITIVE REPORT
JEM © The Rockefeller University Press $15.00
Vol. 204, No. 6, June 11, 2007 1319–1325 www.jem.org/cgi/doi/10.1084/jem.20070432
1319
The complement system is required to mount
an appropriate innate immune response to path-
ogens. It acts by facilitating phagocytosis of
immune complexes and apoptotic cells and by
forming a membrane attack complex resulting
in cell lysis (1). Particles and pathogens in serum
initiate complement activation either through
the classical pathway (CP), mannose-binding
lectin (MBL) pathway, or alternative pathway
(AP; reference 2). Central to complement acti-
vation are the convertases, enzyme complexes
that cleave the substrates C3 and C5 into their
biologically active fragments, C3a, C3b, C5a, and
C5b. The C3bBb dimer and C3bC3bBb mul-
timers form the convertases of the AP and are
required for ampliication of complement acti-
vated through any of the three pathways, whereas
C4bC2a and C3bC4bC2a are convertases of the
CP and MBL pathway.
To prevent unwanted complement activa-
tion, most mammalian cells are equipped with
regulators that block complement ampliication
on host self cells (3). In the absence of these in-
trinsic regulators, serum exposure results in the
generation of complement split product that in
turn facilitates inlammation and tissue damage
(4, 5). Noncellular surfaces that lack intrinsic
complement regulators are therefore especially
prone to complement attack and are fully de-
pendent on protection by soluble complement
regulators in serum. Uncontrolled complement
activation due to the lack of appropriate comple-
ment regulation has been associated with various
chronic inlammatory diseases. Dominant in this
inlammatory cascade are the complement split
products C3a and C5a that function as chemo-
attractant and activators of neutrophils and in-
lammatory macrophages via the C3a and C5a
receptors (6). Properdin, released from neutro-
phils, further ampliies the inlammatory cascade
A novel inhibitor of the alternative pathway
of complement reverses inlammation and
bone destruction in experimental arthritis
Kenneth J. Katschke Jr.,
1
Karim Y. Helmy,
1
Micah Stefek,
2
Hongkang Xi,
1
JianPing Yin,
5
Wyne P. Lee,
1
Peter Gribling,
1
Kai H. Barck,
3
Richard A.D. Carano,
3
Robin E. Taylor,
4
Linda Rangell,
4
Lauri Diehl,
4
Philip E. Hass,
2
Christian Wiesmann,
5
and Menno van Lookeren Campagne
1
1
Department of Immunology,
2
Department of Protein Chemistry,
3
Department of Tumor Biology and Angiogenesis,
4
Department of Pathology, and
5
Department of Protein Engineering, Genentech, Inc., San Francisco, CA 94080
Complement is an important component of the innate and adaptive immune response, yet
complement split products generated through activation of each of the three complement
pathways (classical, alternative, and lectin) can cause inlammation and tissue destruction.
Previous studies have shown that complement activation through the alternative, but not
classical, pathway is required to initiate antibody-induced arthritis in mice, but it is unclear
if the alternative pathway (AP) plays a role in established disease. Previously, we have shown
that human complement receptor of the immunoglobulin superfamily (CRIg) is a selective
inhibitor of the AP of complement. Here, we present the crystal structure of murine CRIg
and, using mutants, provide evidence that the structural requirements for inhibition of the
AP are conserved in human and mouse. A soluble form of CRIg reversed inlammation and
bone loss in two experimental models of arthritis by inhibiting the AP of complement in the
joint. Our data indicate that the AP of complement is not only required for disease induc-
tion, but also disease progression. The extracellular domain of CRIg thus provides a novel
tool to study the effects of inhibiting the AP of complement in established disease and
constitutes a promising therapeutic with selectivity for a single complement pathway.
CORRESPONDENCE
Menno van Lookeren Campagne:
menno@gene.com
The online version of this article contains supplemental material.