Clinical trial NCT03706885 - References

Alzheimer Disease, Early Onset — Efavirenz for Patients With Alzheimer's Disease

Citation(s)

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•   Bretillon L, Lutjohann D, Stahle L, Widhe T, Bindl L, Eggertsen G, Diczfalusy U, Bjorkhem I
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•   Bryleva EY, Rogers MA, Chang CC, Buen F, Harris BT, Rousselet E, Seidah NG, Oddo S, LaFerla FM, Spencer TA, Hickey WF, Chang TY
  ACAT1 gene ablation increases 24(S)-hydroxycholesterol content in the brain and ameliorates amyloid pathology in mice with AD. Proc Natl Acad Sci U S A. 2010 Feb 16;107(7):3081-6. doi: 10.1073/pnas.0913828107. Epub 2010 Jan 26.
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•   Kotti T, Head DD, McKenna CE, Russell DW
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•   Kotti TJ, Ramirez DM, Pfeiffer BE, Huber KM, Russell DW
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•   Lam TN, Hui KH, Chan DP, Lee SS
  Genotype-guided dose adjustment for the use of efavirenz in HIV treatment. J Infect. 2015 Nov;71(5):607-9. doi: 10.1016/j.jinf.2015.07.005. Epub 2015 Jul 18. No abstract available.
•   Leoni V, Caccia C
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•   Lerner AJ, Arnold SE, Maxfield E, Koenig A, Toth ME, Fortin B, Mast N, Trombetta BA, Denker J, Pieper AA, Tatsuoka C, Raghupathy S, Pikuleva IA
  CYP46A1 activation by low-dose efavirenz enhances brain cholesterol metabolism in subjects with early Alzheime
•   Liao WL, Dodder NG, Mast N, Pikuleva IA, Turko IV
  Steroid and protein ligand binding to cytochrome P450 46A1 as assessed by hydrogen-deuterium exchange and mass spectrometry. Biochemistry. 2009 May 19;48(19):4150-8. doi: 10.1021/bi900168m.
•   Lund EG, Guileyardo JM, Russell DW
  cDNA cloning of cholesterol 24-hydroxylase, a mediator of cholesterol homeostasis in the brain. Proc Natl Acad Sci U S A. 1999 Jun 22;96(13):7238-43. doi: 10.1073/pnas.96.13.7238.
•   Lund EG, Xie C, Kotti T, Turley SD, Dietschy JM, Russell DW
  Knockout of the cholesterol 24-hydroxylase gene in mice reveals a brain-specific mechanism of cholesterol turnover. J Biol Chem. 2003 Jun 20;278(25):22980-8. doi: 10.1074/jbc.M303415200. Epub 2003 Apr 9.
•   Lutjohann D, Breuer O, Ahlborg G, Nennesmo I, Siden A, Diczfalusy U, Bjorkhem I
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  Plasma 24S-hydroxycholesterol (cerebrosterol) is increased in Alzheimer and vascular demented patients. J Lipid Res. 2000 Feb;41(2):195-8.
•   Maioli S, Bavner A, Ali Z, Heverin M, Ismail MA, Puerta E, Olin M, Saeed A, Shafaati M, Parini P, Cedazo-Minguez A, Bjorkhem I
  Is it possible to improve memory function by upregulation of the cholesterol 24S-hydroxylase (CYP46A1) in the brain? PLoS One. 2013 Jul 16;8(7):e68534. doi: 10.1371/journal.pone.0068534. Print 2013.
•   Mast N, Andersson U, Nakayama K, Bjorkhem I, Pikuleva IA
  Expression of human cytochrome P450 46A1 in Escherichia coli: effects of N- and C-terminal modifications. Arch Biochem Biophys. 2004 Aug 1;428(1):99-108. doi: 10.1016/j.abb.2004.05.012.
•   Mast N, Charvet C, Pikuleva IA, Stout CD
  Structural basis of drug binding to CYP46A1, an enzyme that controls cholesterol turnover in the brain. J Biol Chem. 2010 Oct 8;285(41):31783-95. doi: 10.1074/jbc.M110.143313. Epub 2010 Jul 28.
•   Mast N, Li Y, Linger M, Clark M, Wiseman J, Pikuleva IA
  Pharmacologic stimulation of cytochrome P450 46A1 and cerebral cholesterol turnover in mice. J Biol Chem. 2014 Feb 7;289(6):3529-38. doi: 10.1074/jbc.M113.532846. Epub 2013 Dec 18.
•   Mast N, Liao WL, Pikuleva IA, Turko IV
  Combined use of mass spectrometry and heterologous expression for identification of membrane-interacting peptides in cytochrome P450 46A1 and NADPH-cytochrome P450 oxidoreductase. Arch Biochem Biophys. 2009 Mar 1;483(1):81-9. doi: 10.1016/j.abb.2009.01.002. Epub 2009 Jan 10.
•   Mast N, Linger M, Clark M, Wiseman J, Stout CD, Pikuleva IA
  In silico and intuitive predictions of CYP46A1 inhibition by marketed drugs with subsequent enzyme crystallization in complex with fluvoxamine. Mol Pharmacol. 2012 Nov;82(5):824-34. doi: 10.1124/mol.112.080424. Epub 2012 Aug 2.
•   Mast N, Norcross R, Andersson U, Shou M, Nakayama K, Bjorkhem I, Pikuleva IA
  Broad substrate specificity of human cytochrome P450 46A1 which initiates cholesterol degradation in the brain. Biochemistry. 2003 Dec 9;42(48):14284-92. doi: 10.1021/bi035512f.
•   Mast N, Reem R, Bederman I, Huang S, DiPatre PL, Bjorkhem I, Pikuleva IA
  Cholestenoic Acid is an important elimination product of cholesterol in the retina: comparison of retinal cholesterol metabolism with that in the brain. Invest Ophthalmol Vis Sci. 2011 Feb 1;52(1):594-603. doi: 10.1167/iovs.10-6021. Print 2011 Jan.
•   Mast N, Saadane A, Valencia-Olvera A, Constans J, Maxfield E, Arakawa H, Li Y, Landreth G, Pikuleva IA
  Cholesterol-metabolizing enzyme cytochrome P450 46A1 as a pharmacologic target for Alzheimer's disease. Neuropharmacology. 2017 Sep 1;123:465-476. doi: 10.1016/j.neuropharm.2017.06.026. Epub 2017 Jun 24.
•   Mast N, White MA, Bjorkhem I, Johnson EF, Stout CD, Pikuleva IA
  Crystal structures of substrate-bound and substrate-free cytochrome P450 46A1, the principal cholesterol hydroxylase in the brain. Proc Natl Acad Sci U S A. 2008 Jul 15;105(28):9546-51. doi: 10.1073/pnas.0803717105. Epub 2008 Jul 9.
•   Mast N, Zheng W, Stout CD, Pikuleva IA
  Antifungal Azoles: Structural Insights into Undesired Tight Binding to Cholesterol-Metabolizing CYP46A1. Mol Pharmacol. 2013 Jul;84(1):86-94. doi: 10.1124/mol.113.085902. Epub 2013 Apr 19.
•   Mast N, Zheng W, Stout CD, Pikuleva IA
  Binding of a cyano- and fluoro-containing drug bicalutamide to cytochrome P450 46A1: unusual features and spectral response. J Biol Chem. 2013 Feb 15;288(7):4613-24. doi: 10.1074/jbc.M112.438754. Epub 2013 Jan 3.
•   Meaney S, Bodin K, Diczfalusy U, Bjorkhem I
  On the rate of translocation in vitro and kinetics in vivo of the major oxysterols in human circulation: critical importance of the position of the oxygen function. J Lipid Res. 2002 Dec;43(12):2130-5. doi: 10.1194/jlr.m200293-jlr200.
•   Morris JC
  The Clinical Dementia Rating (CDR): current version and scoring rules. Neurology. 1993 Nov;43(11):2412-4. doi: 10.1212/wnl.43.11.2412-a. No abstract available.
•   Paul SM, Doherty JJ, Robichaud AJ, Belfort GM, Chow BY, Hammond RS, Crawford DC, Linsenbardt AJ, Shu HJ, Izumi Y, Mennerick SJ, Zorumski CF
  The major brain cholesterol metabolite 24(S)-hydroxycholesterol is a potent allosteric modulator of N-methyl-D-aspartate receptors. J Neurosci. 2013 Oct 30;33(44):17290-300. doi: 10.1523/JNEUROSCI.2619-13.2013.
•   Radhakrishnan A, Sun LP, Kwon HJ, Brown MS, Goldstein JL
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•   Ramirez DM, Andersson S, Russell DW
  Neuronal expression and subcellular localization of cholesterol 24-hydroxylase in the mouse brain. J Comp Neurol. 2008 Apr 10;507(5):1676-93. doi: 10.1002/cne.21605.
•   Russell DW, Halford RW, Ramirez DM, Shah R, Kotti T
  Cholesterol 24-hydroxylase: an enzyme of cholesterol turnover in the brain. Annu Rev Biochem. 2009;78:1017-40. doi: 10.1146/annurev.biochem.78.072407.103859.
•   White MA, Mast N, Bjorkhem I, Johnson EF, Stout CD, Pikuleva IA
  Use of complementary cation and anion heavy-atom salt derivatives to solve the structure of cytochrome P450 46A1. Acta Crystallogr D Biol Crystallogr. 2008 May;64(Pt 5):487-95. doi: 10.1107/S0907444908004046. Epub 2008 Apr 19.

A Proof-of-Concept Clinical Research Study of Efavirenz in Patients With Alzheimer's Disease

Details for clinical trial NCT03706885