POLYMER-COATED RED BLOOD CELL FOR SICKLE CELL DISEASE
Project Number1R01HL065637-01
Contact PI/Project LeaderFISHER, TIMOTHY C
Awardee OrganizationUNIVERSITY OF SOUTHERN CALIFORNIA
Description
Abstract Text
DESCRIPTION: (Investigator's abstract) Although red blood cell (RBC)
transfusion is an essential component in the management of acute complications
of sickle cell disease (SCD), and the recent "STOP" study has demonstrated that
chronic blood transfusion can prevent stroke in high-risk SCD children,
transfusion in SCD has associated problems: 1) high alloimmunization rates (up
to 30 percent) and iron accumulation; 2) limitation of post-transfusion
hematocrit to less than 35 percent to avoid blood hyperviscosity which may
precipitate a vaso-occlusive event. However, we have recently developed a
technique which has the potential to mitigate these problems: covalent bonding
of a thin coating of polyethylene glycol (PEG) or related polymers to the RBC
surface. Results to date indicate that consequent to coating, the RBC surface
is inaccessible to antibodies (i.e., the RBC blood group antigens are "masked")
and RBC interactions, such as RBC aggregation, are minimized; the latter effect
results in greatly reduced low-shear blood viscosity even when the hematocrit
of SS blood is increased with coated RBC.
The ultimate objective of this Research Program is the development of safe and
effective RBC polymer coating methods which achieve antigen masking and
viscosity reduction and which offer therapeutic benefits for sickle cell
disease subjects. Specific aims include: 1) optimizing polymer coating
techniques via evaluating linear, branched, star and dendrimer PEG molecules
and various bonding chemistries and crosslinking strategies; 2) evaluation of
the functional status of polymer-coated RBC in terms of RBC morphology,
rheological behavior (i.e., deformability), membrane transport and oxygen
binding, identification of membrane proteins affected (e.g., C-14 labeled
PEGs), the storage ability of coated-cells, and in vivo survival in mice and
rabbit systems; 3) evaluation of polymer-coated RBC as therapeutic agents in
SCD via in vitro rheologic studies of M and SS RBC mixtures at various
hematocrits and oxygen tensions, and via in vivo flow studies using rat
mesocecum and cat skeletal muscle preparations; 4) evaluation of polymer
coating as a means to prevent alloimmunization and/or to protect transfused RBC
in alloimmunized subjects by utilizing both in vitro (e.g., antibody/complement
binding, complement lysis, monocyte monolayer assay) and in vivo approaches
(e.g., alloimmunized rabbit model, xenotransfusions) methods. An interactive
approach to these aims is proposed; their successful achievement should yield
important new data and improved health care in SCD.
No Sub Projects information available for 1R01HL065637-01
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