In the past year, we have completed a pre-clinical animal study in LCAT K/O mice showing the feasibility of using recombinant LCAT as a therapeutic agent (ref. 2) and have also filed a provisional patent application, with our outside collaborators, on the use of LCAT for modulating HDL levels (ref. 1). In addition, we have shown that low LCAT levels was a biomarker for cardiovascular risk in the general population (ref. 3). In conjunction with our outside collaborators, who have licensed the patent from the NIH, we have completed the pre-clinical toxicology studies and have recently filed an IND to start a clinical trial.

Project Start
Project End
Budget Start
Budget End
Support Year
2
Fiscal Year
2011
Total Cost
$688,623
Indirect Cost
Name
National Heart, Lung, and Blood Institute
Department
Type
DUNS #
City
State
Country
Zip Code
Shamburek, Robert D; Bakker-Arkema, Rebecca; Auerbach, Bruce J et al. (2016) Familial lecithin:cholesterol acyltransferase deficiency: First-in-human treatment with enzyme replacement. J Clin Lipidol 10:356-67
Ossoli, Alice; Neufeld, Edward B; Thacker, Seth G et al. (2016) Lipoprotein X Causes Renal Disease in LCAT Deficiency. PLoS One 11:e0150083
Shamburek, Robert D; Bakker-Arkema, Rebecca; Shamburek, Alexandra M et al. (2016) Safety and Tolerability of ACP-501, a Recombinant Human Lecithin:Cholesterol Acyltransferase, in a Phase 1 Single-Dose Escalation Study. Circ Res 118:73-82
Thacker, Seth G; Rousset, Xavier; Esmail, Safiya et al. (2015) Increased plasma cholesterol esterification by LCAT reduces diet-induced atherosclerosis in SR-BI knockout mice. J Lipid Res 56:1282-95
Stukas, Sophie; Freeman, Lita; Lee, Michael et al. (2014) LCAT deficiency does not impair amyloid metabolism in APP/PS1 mice. J Lipid Res 55:1721-9
Liu, Zheng; Thacker, Seth G; Fernandez-Castillejo, Sara et al. (2014) Synthesis of cholesterol analogues bearing BODIPY fluorophores by Suzuki or Liebeskind-Srogl cross-coupling and evaluation of their potential for visualization of cholesterol pools. Chembiochem 15:2087-96
Vaisman, Boris L; Remaley, Alan T (2013) Measurement of lecithin-cholesterol acyltransferase activity with the use of a Peptide-proteoliposome substrate. Methods Mol Biol 1027:343-52
Hoang, Anh; Drew, Brian G; Low, Hann et al. (2012) Mechanism of cholesterol efflux in humans after infusion of reconstituted high-density lipoprotein. Eur Heart J 33:657-65
Rosenson, Robert S; Brewer Jr, H Bryan; Davidson, W Sean et al. (2012) Cholesterol efflux and atheroprotection: advancing the concept of reverse cholesterol transport. Circulation 125:1905-19
Rosenson, Robert S; Brewer Jr, H Bryan; Chapman, M John et al. (2011) HDL measures, particle heterogeneity, proposed nomenclature, and relation to atherosclerotic cardiovascular events. Clin Chem 57:392-410

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