Targeting farnesyl-transferase as a novel therapeutic strategy for mevalonate kinase deficiency: In vitro and in vivo approaches

Pharmacological Research

Volumn 61, Issue 6, 2010, Pages 506-510

  De Leo, Luigina ^a   Marcuzzi, Annalisa ^a   Decorti, Giuliana ^a   Tommasini, Alberto ^a   Crovella, Sergio ^b,c   Pontillo, Alessandra ^b  

^a UNIVERSITY OF TRIESTE   (Italy)

^b INSTITUTE FOR MATERNAL AND CHILD HEALTH IRCCS BURLO GAROFOLO   (Italy)

^c FEDERAL UNIVERSITY OF PERNAMBUCO   (Brazil)

Author keywords

Auto inflammatory syndromes; Farnesol; Farnesyl transferase inhibitors; Geraniol; Geranylgeraniol; Hereditary periodic fevers; Isoprenoids; Manumycin A; Mevalonate kinase deficiency

Indexed keywords

AMYLOID A PROTEIN; GERANYLGERANYL PYROPHOSPHATE; INTERLEUKIN 1BETA; ISOPRENOID; MANUMYCIN; MANUMYCIN A; MEVALONATE KINASE; PROTEIN FARNESYLTRANSFERASE; UNCLASSIFIED DRUG;

ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIINFLAMMATORY ACTIVITY; ARTICLE; CASE REPORT; CELL ISOLATION; CHILD; CONTROLLED STUDY; CYTOKINE RELEASE; DRUG EFFICACY; DRUG SAFETY; ENZYME STRUCTURE; ENZYME SYNTHESIS; FEMALE; HUMAN; HUMAN CELL; MALE; MEVALONATE KINASE DEFICIENCY; MONOCYTE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL;

ADULT; ANIMALS; ANTI-INFLAMMATORY AGENTS; CELLS, CULTURED; CHILD, PRESCHOOL; ENZYME INHIBITORS; FARNESYLTRANSTRANSFERASE; FEMALE; HUMANS; MALE; MEVALONATE KINASE DEFICIENCY; MEVALONIC ACID; MICE; MICE, INBRED BALB C; MONOCYTES; POLYENES; POLYISOPRENYL PHOSPHATES; POLYUNSATURATED ALKAMIDES; YOUNG ADULT;

EID: 77952887728     PISSN: 10436618     EISSN: 10961186     Source Type: Journal    
DOI: 10.1016/j.phrs.2010.02.012     Document Type: Article

References (13)

1. Drenth J.P., Haagsma C.J., van der Meer J.W. Hyperimmunoglobulinemia D and periodic fever syndrome. The clinical spectrum in a series of 50 patients. International Hyper-IgD Study Group. Medicine (Baltimore) 1994, 73:133-144.
2. Fears R. The contribution of the cholesterol biosynthetic pathway to intermediary metabolism and cell function. Biochem J 1981, 199:1-7.
3. Drenth J.P., van der Meer J.W., Kushner I. Unstimulated peripheral blood mononuclear cells from patients with the hyper-IgD syndrome produce cytokines capable of potent induction of C-reactive protein and serum amyloid A in Hep3B cells. J Immunol 1996, 157:400-404.
4. Frenkel J., Rijkers G.T., Mandey S.H., Buurman S.W., Houten S.M., Wanders R.J., et al. Lack of isoprenoid products raises ex vivo interleukin-1beta secretion in hyperimmunoglobulinemia D and periodic fever syndrome. Arthritis Rheum 2002, 46:2794-2803.
5. Mandey S.H., Kuijk L.M., Frenkel J., Waterham H.R. A role for geranylgeranylation in interleukin-1beta secretion. Arthritis Rheum 2006, 54:3690-3695.
6. Kuijk L.M., Mandey S.H., Schellens I., Waterham H.R., Rijkers G.T., Coffer P.J., et al. Statin synergizes with LPS to induce IL-1beta release by THP-1 cells through activation of caspase-1. Mol Immunol 2008, 45:2158-2165.
7. Braun T., Fenaux P. Farnesyltransferase inhibitors and their potential role in therapy for myelodysplastic syndromes and acute myeloid leukaemia. Br J Haematol 2008, 141:576-586.
8. Kouchi H., Nakamura K., Fushimi K., Sakaguchi M., Miyazaki M., Ohe T., et al. Manumycin A, inhibitor of ras farnesyltransferase, inhibits proliferation and migration of rat vascular smooth muscle cells. Biochem Biophys Res Commun 1999, 264:915-920.
9. Marcuzzi A., Pontillo A., De Leo L., Tommasini A., Decorti G., Not T., et al. Natural isoprenoids are able to reduce inflammation in a mouse model of mevalonate kinase deficiency. Pediatr Res 2008, 64:177-182.
10. Xue X., Lai K.T., Huang J.F., Gu Y., Karlsson L., Fourie A. Anti-inflammatory activity in vitro and in vivo of the protein farnesyltransferase inhibitor tipifarnib. J Pharmacol Exp Ther 2006, 317:53-60.
11. Crick D.C., Andres D.A., Waechter C.J. Farnesol is utilized for protein isoprenylation and the biosynthesis of cholesterol in mammalian cells. Biochem Biophys Res Commun 1995, 211:590-599.
12. Houten S.M., Wanders R.J., Waterham H.R. Biochemical and genetic aspects of mevalonate kinase and its deficiency. Biochim Biophys Acta 2000, 1529:19-32.
13. Basso A.D., Mirza A., Liu G., Long B.J., Bishop W.R., Kirschmeier P. The farnesyl transferase inhibitor (FTI) SCH66336 (lonafarnib) inhibits Rheb farnesylation and mTOR signaling. Role in FTI enhancement of taxane and tamoxifen anti-tumor activity. J Biol Chem 2005, 280:31101-31108.