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Defibrotide
Is the sodium salt of a complex mixture of single-stranded
oligodeoxyribonucleotides derived from porcine mucosal DNA. It has been shown
that defibrotide has protective effects on vascular endothelial cells,
particularly those of small vessels. It has extensive beneficial
pharmacological effects owing to its antithrombotic, anti-inflammatory and
antiischemic properties.
Recent clinical trials of defibrotide for the treatment of severe
Hepatic
Veno-Occlusive Disease (VOD) have indicated both efficacy and lack of
significant toxicity. Hepatic VOD is a potentially devastating complication of
both allogeneic and autologous stem cell transplantation. Severe VOD usually
occurs very early in the course of transplantation and the mortality rate of
these cases is at least 90%.
Other recent preclinical studies have demonstrated that defibrotide
used in
conjunction with Granulocyte Colony-Stimulating Factor (rhG-CSF) significantly
increases the number of Peripheral Blood Progenitor Cells (Stem cells). The
benefit of this increase in stem cells may be crucial for a variety of clinical
indications, including graft engineering procedures and gene therapy programs.
The observed effects of Defibrotide in both preclinical and
clinical trials have
opened up new avenues of research for this drug in areas of unmet need such as
cancer, inflammation, and thrombosis. Gentium's expert R&D team are
dedicated in the investigation of Defibrotide's wide-ranging pharmacological
activities.
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