In the past year, we completed the analysis of samples from a Phase I clinical trial of recombinant LCAT (rLCAT), which we completed in the fall of 2012. The study was done at the NIH and was a single dose escalation study in 18 subjects. There was no significant adverse events and the the recombinant LCAT behaved like expected based on animal studies in regard to its ability to increase HDL levels. We were also granted approval for a single patient IND for the treatment of a patient with Familial LCAT Deficiency (FLD) with rLCAT. The rLCAT treatment restored cholesteryl ester levels to near normal levels and above threshold necessary to prevent LpX formation, the abnormal lipoprotein particle that accumulates in FLD and causes renal disease. The rLCAT treatment also corrected the other lipoprotein abnormalities in this disorder, such as low HDL, which may contribute to cardiovascular disease.

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National Heart, Lung, and Blood Institute
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Stukas, Sophie; Freeman, Lita; Lee, Michael et al. (2014) LCAT deficiency does not impair amyloid metabolism in APP/PS1 mice. J Lipid Res 55:1721-1729
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