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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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
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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
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