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Research Laboratories

 

Raphael A. Clynes, M.D., Ph.D.

Assistant Professor of Medicine, Division of Pulmonary, Allergy and Critical Care Medicine

 

 

 

 

Laboratory Address

Columbia University, College of Physicians & Surgeons

630 W. 168th Street, P&S 8-510, New York, NY, 10032

 

Phone 212-305-5289

Fax 212-305-1392

 

 

 

The research focus of the laboratory is to define the mechanisms of antibody-mediated cytotoxicity as applied to both tumor immunity and immune complex disease. We have shown using genetic studies in mice that the cellular receptors for IgG (Fc receptors) modulate the host response triggered by immune complexes and cellular bound antibody. The human and mouse IgG Fc receptor (FcgammaR) family are highly conserved and all murine FcgammaR family members have homologous counterparts in the human system. Both systems share both activating and inhibitory receptors for IgG. The activating receptors for IgG, FcgammaRI and FcgammaRIII both require an additional chain, the gamma subunit, for receptor signaling and assembly. The third receptor for IgG, FcgammaRII, is an inhibitory receptor; receptor coligation of FcgammaRII to either activating receptor FcgammaRI or III, results in down-modulation of the activation signals. The co-expression of both activating and inhibitory Fc receptors on the membrane of hematopoietic cells suggests that the coordinated regulation of these receptors alters the threshold of IC-mediated activation thereby setting the state of cellular IC-responsiveness. Our studies of IgG-triggered effector responses in gene targeted mice have revealed this balanced regulation of IC-effector responses by the opposing effects of genetic disruption of the activating and inhibitory FcgammaR pathways.

 

In models of autoimmunity, mice deficient in the activating Fc receptors are protected from immune complex-triggered inflammatory responses whereas mice lacking inhibitory Fc receptors exhibit dramatically enhanced inflammatory responses. Furthermore the protective anti-tumor responses provided by anti-melanoma, lymphoma and breast adenocarcinoma monoclonal antibodies require Fc receptor interactions in vivo whereas the biological tumorocidal activity of these antibodies are more than 10-fold enhanced in mice lacking inhibitory Fc receptors. These studies indicate that the threshold of activation triggered by IgG immune complexes is determined by the modulated expression of both activating and inhibitory Fc receptors on cellular effectors. We are interested in determining the identity of these Fc receptor-bearing effector cells that are responsible for both IgG mediated anti-tumor effects and autoimmune inflammation as well as the molecular mediators employed by the effector cell arsenal which lead to target cell-death.

 

Antibody-Mediated Tumor Immunity

 

Effective immunity against neoplastic disease requires the specific recognition of tumor antigens coupled with the activation of the effector cytotoxic response. The understanding of the common underlying cytotoxic mechanisms elicited by these approaches would provide the basis for attempts to improve the efficacy of tumor immunotherapeutics.

 

We are interested in defining the molecular mechanisms of antibody-mediated cytotoxicity (ADCC) by identifying the roles of specific inhibitory and activating cellular receptors for IgG, the effector cell type responsible for ADCC in vivo and the molecular mechanisms employed by these effectors to kill tumor target cells. Recently we have demonstrated that the disruption of the activation FcR pathway was sufficient to completely abrogate tumor protection afforded by several monoclonal antibody anti-tumor antibodies including 1) anti-melanoma monoclonal and polyclonal antibodies in a mouse melanoma pulmonary metastases model and 2) anti-HER2/neu and anti-CD20 mAbs in xenograft nude mouse models of human breast cancer and lymphoma. Conversely in mice that lack the inhibitory receptor for IgG the efficacy of antitumor antibodies is dramatically enhanced. Most effector cells (including neutrophils and monocytes) express both activating and inhibitory Fc Receptors. Thus the threshold of effector cell activation by IgG is determined by the balanced ouput of these two opposing signaling pathways. These data demonstrate an unexpected and critical role for activating/inhibitory FcgammaRs in modulating tumor cytotoxicity in vivo and suggest that enhancement of FcgammaR-mediated an body-dependent cellular cytotoxicity by inflammatory cells is a key step in the development of effective antibody-mediated immunotherapeutics.

 

Immune Complex Disease

 

The immune complex triggered inflammatory response is the pathogenic mechanism of tissue injury in a number of autoimmune diseases including arthritis, vasculitis and glomerulonephritis (GN). In particular the deposition of circulating DNA-antiDNA immune complexes leads to the development of vascular and glomerular lesions in SLE. The "Lupus-prone" NZB/NZW mouse model shares several pathogenic and genetic similarities with human S.L.E. including a female bias, and the appearance of IgG containing pathogenic anti-DNA autoantibodies which heralds the onset of severe rapidly progressive GN. The disease process may be separated into two processes: the afferent limb of autoantibody production with concomitant generation and tissue deposition of circulating immune complexes and the subsequent effector limb of the inflammatory response elicited by ICs. Since immune complex tissue deposits avidly fix complement, the effector responses may be triggered by either cellular Fc receptors or by complement mediated pathways. The importance of the Fc receptor effector pathway in modulating immune complex inflammation has been established in mice bearing targeted disruptions in FcR genes. In these gamma -/- mice the afferent limb of autoimmunity is intact but the subsequent inflammatory response is uncoupled. Thus gamma -/- mice are protected from autoimmune injury in models of autoimmune hemolytic anemia, autoimmune thrombocytopenia, and from immune complex injury in the lung, kidney and skin. Both NZB/NZW gamma -/- and +/+ mice develop autoantobodies with glomerular deposition of immune complexes but the uncoupling of the effector response prevents the development of fatal nephritis in g -/-. These studies challenge the prevailing view that complement components are sufficient to initiate IC-mediated inflammatory responses and argue for the development of FcR targeted therapeutics in the treatment of immune complex mediated disease.

Immune complex injury may result from activation of resident tissue inflammatory cells and/or the recruitment of circulating monocytes or neutrophils to sites of deposition. In the kidney, activation and proliferation of the resident mesangial cell is a hallmark of immune complex inflammatory disease. Because of this circularity of the proinflammatory cytokine network it is unclear which cell(s) are critical to the initiation of the inflammatory cascade. Since Fc receptors are expressed on most leukocytes, including monocytes and neutrophils, and may also be expressed on resident mesangial cells, a number of potential initiation pathways are possible. The identification of specific FcR-bearing effector cells responsible for the initiation of this clinically relevant IC-mediated disease will have general implications for the understanding of mechanisms underlying systemic autoimmune disease.

 


 

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