Development and Pharmacology of Novel Lipidic rAHF
Project Number5R01HL070227-06
Contact PI/Project LeaderBALU-IYER, SATHY VENKAT
Awardee OrganizationSTATE UNIVERSITY OF NEW YORK AT BUFFALO
Description
Abstract Text
Advances in protein engineering have led to the development of proteins as therapeutic agents. However, a
common complication is the reduction of efficacy due to antibody response. Factors that influence antibody
response include protein aggregation, immunogenic sequences within the protein and the frequency of
administration. The broad objective of this proposal is to improve therapeutic efficacy of biopharmaceuticals
by developing lipid-protein complexes that will reduce immunogenicity and decrease the clearance of the
protein, thereby reducing frequency of administration. Factor VIII (FVIII) offers an excellent opportunity to
investigate such approaches, as the administration of exogenous FVIII leads to development of inhibitory
antibody responses in 15-30% of Hemophilia A patients, complicating replacement therapy. The long term
goal of the project is to develop lipidic complexes of FVIII that positively modulate immunogenicity and
clearance. During the previous project period, rational approaches led to the development of FVIII-
Phosphoserine (PS)complexes that showed reduction in immune response against the protein following its
injection in Hemophilia A mice and improved physical stability. The molecular interaction of PS with FVIII and
Calcium ions was exploited to develop lipid based nano/micro particulates including liposomes and novel
condensed and nano-cochleate structures for FVIII delivery. In this proposal, we propose to investigate (1)
the mechanism of reduction in immune response mediated by FVIM-PS complexes (2) the effect of low
density receptor related protein and von Willebrand factor mediated clearance of FVIII and FVIII-PS
complexes and (3) the effect of antibody response on clearance of FVIII and FVIII-PS complexes. The
studies aimed at understanding the pharmacology of FVIII-PS complexes will be carried out in Hemophilia A
mice model and in vitro with antigen presenting cells such as dendritic cells and T-cells. We propose to
investigate key steps in the processing of protein antigen by the immune system in general, which include
uptake and processing of FVIII by antigen presenting cells and subsequent presentation and expansion of T-
cells. We will investigate the role of PS in modulating the immunogenicity of FVIII (Specific Aim 1). The
pharmacokinetic properties of FVIII are complex due to intrinsic protein binding and we will investigate the
disposition of FVIII and FVIII-PS complexes mediated by liver metabolism and immune system. In specific
aim 2, we will investigate the effect of PS binding and lipid molecular assemblies on pharmacokinetic
parameters such as half-life, area under the curve and clearance. Finally, in specific aim 3, we will
investigate the effect of antibody response and disposition of FVIII. The results obtained from these studies
will be useful to develop optimal dosing and efficient management of the disease and therapy.
Public Health Relevance Statement
Data not available.
NIH Spending Category
Biotechnology
Project Terms
A MouseAbbreviationsAntibodiesAntibody FormationAntigen-Presenting CellsAntigensArea Under CurveBindingBinding SitesBlood CirculationC2 DomainCalcium ionCell Culture TechniquesCholineCircular DichroismComplexComplicationDendritic CellsDevelopmentDimyristoylphosphatidylcholineDisease ManagementDoseDrug FormulationsDrug KineticsDrug or chemical Tissue DistributionEncapsulatedEpitopesEthanolaminesEthylene GlycolsExogenous FactorsFactor VIIIFrequenciesGenerationsGoalsHalf-LifeHemophilia AImmune responseImmune systemIn VitroInflammatory ResponseInjection of therapeutic agentKnockout MiceLDL-Receptor Related Protein 1LeadLipid BindingLipidsLipopolysaccharidesLiposomesMediatingMicellesModificationMolecularMolecular Sieve ChromatographyMusParticle SizeParticulatePatientsPharmacologic SubstancePharmacologyPhosphatidic AcidPhospholipidsPhosphoserinePlasmaPlayPreparationProcessPropertyProtein BindingProtein EngineeringProteinsRecombinantsReplacement TherapyResearch PersonnelReticuloendothelial SystemRoleSerineStructureSurfaceT-Cell ActivationT-LymphocyteTestingTherapeuticTherapeutic AgentsTime FactorsTreatment Efficacybasecytokinedensitydesigndioleoyl-N-(monomethoxypolyethylene glycol succinyl)phosphatidylethanolamineethanolamineethylene glycolimmunogenicimmunogenicityimprovedin vivoinhibitor/antagonistliver metabolismmolecular assembly/self assemblymouse modelnanonovelparticlepreventprogramsprotein aggregationreceptorreceptor densityrecombinant antihemophilic factor VIIIresearch studytherapeutic proteinuptakevon Willebrand Factor
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