Anti-fibroblast growth factor 23 antibody therapy

Current Opinion in Nephrology and Hypertension

Volumn 23, Issue 4, 2014, Pages 346-351

  Fukumoto, Seiji ^a  

^a UNIVERSITY OF TOKYO HOSPITAL   (Japan)

Author keywords

Extracellular signal regulated kinase; FAM20C; Fibroblast growth factor receptor; Hypophosphatemia; Osteomalacia; Rickets

Indexed keywords

CALCITRIOL; FIBROBLAST GROWTH FACTOR 23; FIBROBLAST GROWTH FACTOR RECEPTOR; MITOGEN ACTIVATED PROTEIN KINASE; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY FIBROBLAST GROWTH FACTOR 23; UNCLASSIFIED DRUG; CASEIN KINASE I; FAM20C PROTEIN, HUMAN; FIBROBLAST GROWTH FACTOR; IMMUNOLOGIC FACTOR; PHOSPHATE; SCLEROPROTEIN;

CARBOXY TERMINAL SEQUENCE; DRUG SAFETY; FAM20C GENE; FGF23 GENE; GENE; GENE MUTATION; HRAS GENE; HUMAN; HYPOPHOSPHATEMIA; IN VITRO STUDY; IN VIVO STUDY; MOLECULAR CLONING; NONHUMAN; ONCOGENE N RAS; ONCOGENIC OSTEOMALACIA; PHASE 1 CLINICAL TRIAL (TOPIC); PHOSPHATE BLOOD LEVEL; PHOSPHATE METABOLISM; PRIORITY JOURNAL; REVIEW; X LINKED HYPOPHOSPHATEMIC RICKETS; ANIMAL; ANTAGONISTS AND INHIBITORS; BLOOD; COMPLICATION; FAMILIAL HYPOPHOSPHATEMIC RICKETS; GENETICS; IMMUNOLOGY; METABOLISM; NEOPLASM; OSTEOMALACIA;

ANIMALS; ANTIBODIES, MONOCLONAL, HUMANIZED; CASEIN KINASE I; EXTRACELLULAR MATRIX PROTEINS; FAMILIAL HYPOPHOSPHATEMIC RICKETS; FIBROBLAST GROWTH FACTORS; HUMANS; HYPOPHOSPHATEMIA; IMMUNOLOGIC FACTORS; NEOPLASMS; OSTEOMALACIA; PHOSPHATES;

EID: 84902287981     PISSN: 10624821     EISSN: 14736543     Source Type: Journal    
DOI: 10.1097/01.mnh.0000447012.98357.da     Document Type: Review

References (39)

1. ADHR Consortium. Autosomal dominant hypophosphataemic rickets is associated with mutations in FGF23. Nat Genet 2000; 26:345-348.
2. Shimada T, Mizutani S, Muto T, et al. Cloning and characterization of FGF23 as a causative factor of tumor-induced osteomalacia. Proc Natl Acad Sci U S A 2001; 98:6500-6505.
3. Hughes MR, Brumbaugh PF, Hussler MR, et al. Regulation of serum 1alpha, 25-dihydroxyvitamin D3 by calcium and phosphate in the rat. Science 1975; 190:578-580.
4. Shimada T, Hasegawa H, Yamazaki Y, et al. FGF-23 is a potent regulator of vitamin D metabolism and phosphate homeostasis. J Bone Miner Res 2004; 19:429-435.
5. Kurosu H, Ogawa Y, Miyoshi M, et al. Regulation of fibroblast growth factor- 23 signaling by klotho. J Biol Chem 2006; 281:6120-6123.
6. Urakawa I, Yamazaki Y, Shimada T, et al. Klotho converts canonical FGF receptor into a specific receptor for FGF23. Nature 2006; 444:770-774.
7. The HYP Consortium. A gene (PEX) with homologies to endopeptidases is mutated in patients with X-linked hypophosphatemic rickets. NatGenet 1995; 11:130-136.
8. Jonsson KB, Zahradnik R, Larsson T, et al. Fibroblast growth factor 23 in oncogenic osteomalacia and X-linked hypophosphatemia. N Engl J Med 2003; 348:1656-1663.
9. Yamazaki Y, Okazaki R, Shibata M, et al. Increased circulatory level of biologically active full-length FGF-23 in patients with hypophosphatemic rickets/osteomalacia. J Clin Endocrinol Metab 2002; 87:4957-4960.
10. Liu S, Guo R, Simpson LG, et al. Regulation of fibroblastic growth factor 23 expression but not degradation by PHEX. J Biol Chem 2003; 278:37419-37426.
11. Sitara D, Razzaque MS, Hesse M, et al. Homozygous ablation of fibroblast growth factor-23 results in hyperphosphatemia and impaired skeletogenesis, and reverses hypophosphatemia in Phex-deficient mice. Matrix Biol 2004; 23:421-432.
12. Yuan B, Takaiwa M, Clemens TL, et al. Aberrant Phex function in osteoblasts and osteocytes alone underlies murine X-linked hypophosphatemia. J Clin Invest 2008; 118:722-734.
13. Fukumoto S, Shimizu Y. Fibroblast growth factor 23 as a phosphotropic hormone and beyond. J Bone Miner Metab 2011; 29:507-514.
14. Tagliabracci VS, Engel JL, Wen J, et al. Secreted kinase phosphorylates extracellular proteins that regulate biomineralization. Science 2012; 336:1150-1153.
15. Simpson MA, Hsu R, Keir LS, et al. Mutations in FAM20C are associated with lethal osteosclerotic bone dysplasia (Raine syndrome), highlighting a crucial molecule in bone development. Am J Hum Genet 2007; 81:906-912.
16. Simpson MA, Scheuerle A, Hurst J, et al. Mutations in FAM20C also identified in nonlethal osteosclerotic bone dysplasia. Clin Genet 2009; 75:271-276.
17. Wang X, Wang S, Li C, et al. Inactivation of a novel FGF23 regulator, FAM20C, leads to hypophosphatemic rickets in mice. PLoS Genet 2012; 8:e1002708.
18. Rafaelsen SH, Raeder H, Fagerheim AK, et al. Exome sequencing reveals FAM20c mutations associated with fibroblast growth factor 23-related hypophosphatemia, dental anomalies, and ectopic calcification. J Bone Miner Res 2013; 28:1378-1385.
19. Feng JQ, Ward LM, Liu S, et al. Loss of DMP1 causes rickets and osteomalacia and identifies a role for osteocytes in mineral metabolism. Nat Genet 2006; 38:1310-1315.
20. Lorenz-Depiereux B, Bastepe M, Benet-Pages A, et al. DMP1 mutations in autosomal recessive hypophosphatemia implicate a bone matrix protein in the regulation of phosphate homeostasis. Nat Genet 2006; 38:1248-1250.
21. Hoffman WH, Jueppner HW, Deyoung BR, et al. Elevated fibroblast growth factor-23 in hypophosphatemic linear nevus sebaceous syndrome. Am J Med Genet A 2005; 134:233-236.
22. Avitan-Hersh E, Tatur S, Indelman M, et al. Postzygotic HRAS mutation causing both keratinocytic epidermal nevus and thymoma and associated with bone dysplasia and hypophosphatemia due to elevated FGF23. J Clin Endocrinol Metab 2014; 99:E132-E136.
23. Lim YH, Ovejero D, Sugarman JS, et al. Multilineage somatic activating mutations in HRAS and NRAS cause mosaic cutaneous and skeletal lesions, elevated FGF23 and hypophosphatemia. Hum Mol Genet 2014; 23:397-407.
24. Folpe AL, Fanburg-Smith JC, Billings SD, et al.Most osteomalacia- Associated mesenchymal tumors are a single histopathologic entity: an analysis of 32 cases and a comprehensive review of the literature. Am J Surg Pathol 2004; 28:1-30.
25. Jin X, Jing H, Li F, Zhuang H. Osteomalacia-inducing renal clear cell carcinoma uncovered by 99mTc-Hydrazinonicotinyl-Tyr3-octreotide (99mTc-HYNICTOC) scintigraphy. Clin Nucl Med 2013; 38:922-924.
26. Leaf DE, Pereira RC, Bazari H, Juppner H. Oncogenic osteomalacia due to FGF23-expressing colon adenocarcinoma. J Clin Endocrinol Metab 2013; 98:887-891.
27. Xie Y, Li HZ. Oncogenic osteomalacia caused by renal cell carcinoma. J Clin Endocrinol Metab 2013; 98:4597-4598.
28. Carpenter TO, Imel EA, Holm IA, et al. A clinicians guide to X-linked hypophosphatemia. J Bone Miner Res 2011; 26:1381-1388.
29. Goetz R, Nakada Y, Hu MC, et al. Isolated C-Terminal tail of FGF23 alleviates hypophosphatemia by inhibiting FGF23-FGFR-Klotho complex formation. Proc Natl Acad Sci U S A 2010; 107:407-412.
30. Wohrle S, Bonny O, Beluch N, et al. FGF receptors control vitamin D and phosphate homeostasis by mediating renal FGF-23 signaling and regulating FGF-23 expression in bone. J Bone Miner Res 2011; 26:2486-2497.
31. Wohrle S, Henninger C, Bonny O, et al. Pharmacological inhibition of fibroblast growth factor (FGF) receptor signaling ameliorates FGF23- mediated hypophosphatemic rickets. J Bone Miner Res 2013; 28:899-911.
32. Ranch D, Zhang MY, Portale AA, Perwad F. Fibroblast growth factor 23 regulates renal 1,25-dihydroxyvitamin D and phosphate metabolism via the MAP kinase signaling pathway inHypmice. JBone MinerRes2011;26:1883-1890.
33. Zhang MY, Ranch D, Pereira RC, et al. Chronic inhibition of ERK1/2 signaling improves disordered bone and mineral metabolism in hypophosphatemic (Hyp) mice. Endocrinology 2012; 153:1806-1816.
34. Yamazaki Y, Tamada T, Kasai N, et al. Anti-FGF23 neutralizing antibodies demonstrate the physiological role and structural features of FGF23. J Bone Miner Res 2008; 23:1509-1518.
35. Kuro-o M, Matsumura Y, Aizawa H, et al. Mutation of the mouse klotho gene leads to a syndrome resembling ageing. Nature 1997; 390:45-51.
36. Shimada T, Kakitani M, Yamazaki Y, et al. Targeted ablation of Fgf23 demonstrates an essential physiological role of FGF-23 in phosphate and vitamin D metabolism. J Clin Invest 2004; 113:561-568.
37. Aono Y, Yamazaki Y, Yasutake J, et al. Therapeutic effects of anti-FGF23 antibodies in hypophosphatemic rickets/osteomalacia. J Bone Miner Res 2009; 24:1879-1888.
38. Aono Y, Hasegawa H, Yamazaki Y, et al. Anti-FGF23 neutralizing antibodies ameliorate muscle weakness and decreased spontaneous movement of Hyp mice. J Bone Miner Res 2011; 26:803-810.
39. Carpenter TO, Imel E, Ruppe M, et al. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. J Clin Invest 2014; 124:1587-1597.