Von Willebrand Disease Clinical Trial
Official title:
Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)
Von Willebrand disease is an inherited bleeding disorder that impacts the blood's ability to
clot properly. Von Willebrand disease is cause by the lack or not working substance in the
blood known as Von Willebrand factor.
Current therapy for Von Willebrand disease includes desmopressin acetate (DDAVP) and /or
VWF/FVIII concentrates. Patients with severe Von Willebrand disease face a lifetime of
weekly treatments and mounting medical bills. Gene therapy could help these patients improve
their quality of life by providing the missing factors necessary for the blood's ability to
clot properly.
The gene transfer options being studied include naked DNA, viral gene transfer vectors
encoding Von Willebrand factor transgenes, and ex vivo cell therapy. The latter involves
transplantation of the patient's own cells modified with a corrected copy of the defective
gene. Human blood outgrowth endothelial cells (BOEC) display all the properties needed for
successful ex vivo cell therapy. We plan to obtain blood samples from normal research
subjects and patients with Von Willebrand Disease in order to isolate blood outgrowth
endothelial cells (BOEC) from peripheral blood, and develop a ex vivo gene therapy for Von
Willebrand Disease.
Aim: The purpose of this protocol is to obtain blood samples from normal research subjects
and patients with Von Willebrand disease in order to isolate blood outgrowth endothelial
cells (BOEC) from peripheral blood, and develop a ex vivo gene transfer for Von Willebrand
disease.
Experimental procedure. This protocol is designed to gather approximately 90 ml (6
tablespoons) of blood from normal individuals without Von Willebrand disease and individuals
with known Von Willebrand disease. A total of 20 normal individuals and 20 Von Willebrand
disease patients will be enrolled into the study.
Studies to be performed on the blood samples:
Each blood draw will require 10 large blue/black capped (Na Citrate) vacutainer (16x125mm,
BD# 362761, Cell Preparation Tubes (CPT) to be filled from each volunteer. All the blood
will be used for cell processing.
In initial proof-of-concept experiments, BOEC lines will be established from the peripheral
blood of normal individuals and research subjects with Von Willebrand disease. Following
culture and phenotypic characterization, they will be transduced with a lentivirus gene
transfer vector expressing a reporter gene or wild-type Von Willebrand factor. After
demonstrating successful transduction, the next step will be to write an animal protocol
that will introduce the modified cells into immunocompromised mice and monitor the mice for
stable implantation.
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