Dramatic beneficial effect of interleukin-1 inhibitor treatment in patients with familial Mediterranean fever complicated with amyloidosis and renal failure

Stojanovic, Katia Stankovic; Delmas, Yahsou; Torres, Pablo; Peltier, Julie; Pellé, Gaëlle; Jéru, Isabelle; Colombat, Magali; Grateau, Gilles

doi:10.1093/ndt/gfr528

Cited by 92 publications

(39 citation statements)

References 14 publications

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“…Anakinra is also given to other inflammatory or autoinflammatory diseases off-label. Familial Mediterranean Fever (FMF) is a hereditary chronic inflammatory disease which IL-1 plays a major role in, and blocking IL-1 reduces the symptoms [61, 62]. Anakinra was also shown to be effective in the case of nonhereditary chronic systemic inflammatory diseases like the adult-onset Still disease [63, 64], which involves arthritis, fever, and systemic inflammation or the childhood version—systemic-onset juvenile idiopathic arthritis (SJIA) [65–67].…”

Section: Anti-inflammatory Cytokine Biologicsmentioning

confidence: 99%

Biologics for Targeting Inflammatory Cytokines, Clinical Uses, and Limitations

Rider

Carmi

Bar‐Sela

2016

International Journal of Cell Biology

169

111

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Proinflammatory cytokines are potent mediators of numerous biological processes and are tightly regulated in the body. Chronic uncontrolled levels of such cytokines can initiate and derive many pathologies, including incidences of autoimmunity and cancer. Therefore, therapies that regulate the activity of inflammatory cytokines, either by supplementation of anti-inflammatory recombinant cytokines or by neutralizing them by using blocking antibodies, have been extensively used over the past decades. Over the past few years, new innovative biological agents for blocking and regulating cytokine activities have emerged. Here, we review some of the most recent approaches of cytokine targeting, focusing on anti-TNF antibodies or recombinant TNF decoy receptor, recombinant IL-1 receptor antagonist (IL-1Ra) and anti-IL-1 antibodies, anti-IL-6 receptor antibodies, and TH17 targeting antibodies. We discuss their effects as biologic drugs, as evaluated in numerous clinical trials, and highlight their therapeutic potential as well as emphasize their inherent limitations and clinical risks. We suggest that while systemic blocking of proinflammatory cytokines using biological agents can ameliorate disease pathogenesis and progression, it may also abrogate the hosts defense against infections. Moreover, we outline the rational need to develop new therapies, which block inflammatory cytokines only at sites of inflammation, while enabling their function systemically.

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Section: Anti-inflammatory Cytokine Biologicsmentioning

confidence: 99%

Biologics for Targeting Inflammatory Cytokines, Clinical Uses, and Limitations

Rider

Carmi

Bar‐Sela

2016

International Journal of Cell Biology

169

111

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“…Thus, better treatments for rheumatoid arthritis and disorders of pyrin-associated pathways (e.g., familial Mediterranean fever and related disorders) utilizing biologics targeting either cytokines or their receptors have markedly reduced the incidence of AA as a consequence of these conditions. [122][123][124] The apparent uniqueness of the process of amyloidogenesis related to TTR deposition, in which the monomer aggregation precursor concentration is increased in the absence of increased synthesis or aberrant processing (J. Kelly, this volume), has allowed TTR-specific approaches to therapy and possible prophylaxis. Since 1990, the standard therapy for this disease has been liver transplantation.…”

Section: The Application Of Molecular Insights To Therapy (1975 Onward)mentioning

confidence: 99%

A Molecular History of the Amyloidoses

Buxbaum

Linke²

2012

Journal of Molecular Biology

139

121

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“…In a very recent period of time, it has been reported that it is effective on the treatment of several human inflammatory disorders, such as Mevalonate kinase deficiency (MKD)[18], systemic juvenile idiopathic arthritis (SJIA) [19–21], TNF receptor-associated periodic syndrome (TRAPS) [22], refractory chronic tophaceous gout [23], and refractory adult-onset Still’s disease [13], Schnitzler syndrome [24], cryopyrin-associated periodic syndromes [25], refractory Sweet’s syndrome [26], autoinflammatory Muckle–Wells syndrome [27], persistent knee effusion [28], familial Mediterranean fever [29], Castleman’s disease [30], and arthrofibrosis [31]. …”

Section: Il-1 Receptor Antagonist Human Diseases and Pharmaceutimentioning

confidence: 99%

Anakinra as an interleukin 1 receptor antagonist, complicated genetics and molecular impacts- from the point of view of mouse genomics

Cao

Jiao

Wang

et al. 2012

International Immunopharmacology

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IL-1 receptor antagonist (IL-1rn) is a protein that binds to IL-1 receptors (IL-1r1) and inhibits the binding of IL-1α and IL-1β. In recent years, IL-1rn has been implicated to be associated with many human health problems. The effects of treatment of several inflammatory disorders with anakinra, which is an interleukin-1 (IL-1) receptor antagonist, have also been reported. Both positive and negative effects have been described. In this review, we systematically analyzed the expression, correlation, and regulation of IL-1rn and its 13 partner genes using available gene expression profiles from a variety of tissues in a well known transcriptome database, Genenetwork. The 13 partner genes include IL-1r1, IL-1β, IL-1α, Myd88, Irak1, Irak2, Irak4, Traf6, Tlr4, IL-1rap, Ikbkap, Nfkb1, and Nfkb2. Gene expression profiles are from 10 tissues including spleen, kidney, lung, whole brain, eye, prefrontal cortex, cerebellum, hippocampus, striatum, and nucleus accumbens. Our analysis indicated that the interactions among IL-1rn and its partner are complex and different from tissues to tissues, suggesting a broad spectrum of the effect of IL-1rn on biological and metabolic pathways. Transcripts and protein sequences resulted from different splicing, interaction with genomic background of individuals, and environmental factors affect function of IL-1rn. At present, our knowledge on the function of IL-1rn and its partner in various tissues or organs is very limited. The long term and extended effect of anakinra on human health needs further investigations. In the future, targeted sequences or oligos of Il-1rn might be ideal for therapeutic application with less toxic and more specific in the treatment of specific disease. Detailed study on the molecular function of IL-1rn and its interaction with other genes and environmental factors is essential for development therapeutic application using IL-1rn.

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Dramatic beneficial effect of interleukin-1 inhibitor treatment in patients with familial Mediterranean fever complicated with amyloidosis and renal failure

Cited by 92 publications

References 14 publications

Biologics for Targeting Inflammatory Cytokines, Clinical Uses, and Limitations

Biologics for Targeting Inflammatory Cytokines, Clinical Uses, and Limitations

A Molecular History of the Amyloidoses

Anakinra as an interleukin 1 receptor antagonist, complicated genetics and molecular impacts- from the point of view of mouse genomics

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