Healthcare Industry News: liver failure
News Release - January 20, 2006
Gentium Initiates Phase II/III Pediatric Trial with Defibrotide to Prevent Veno-Occlusive DiseaseEuropean Group for Blood and Marrow Transplantation to Co-Sponsor Study at 30 European and Israeli Clinical Sites
VILLA GUARDIA (COMO), Italy--(HSMN NewsFeed)--Jan. 20, 2006--Gentium S.p.A. (AMEX:GNT ; the "Company") today announced the initiation of a Phase II/III trial with Defibrotide to prevent Veno-Occlusive Disease ("VOD"), a complication of bone marrow and stem cell transplantation (SCT), in pediatric patients. The randomized study will include 270 pediatric patients undergoing SCT at 30 clinical sites in Europe and Israel and will evaluate the ability of Defibrotide to prevent VOD. Secondary endpoints are measuring the severity of VOD and the occurrence of transplant-associated microangiopathy in each group. The European Group for Blood and Marrow Transplantation (EBMT) is co-sponsoring the study with additional support from the Deutsche Krebshilfe (German Cancer Aid). The principal investigator is Selim Corbacioglu, M.D., Dept of Pediatrics - University of Ulm, Germany.
Certain chemotherapy and radiation therapies such as those used in stem cell transplantation can damage cells of the blood vessels and produce VOD, a blockage of the small veins of the liver which leads to damage of the liver cells. Approximately 20% of patients who undergo SCT develop VOD and approximately one-third of these patients progress to multiple organ failure (Severe VOD). Nearly 80% of Severe VOD patients die within three months. There are currently no approved therapies to treat or prevent VOD.
Laura Ferro, M.D., president and chief executive officer of Gentium, said, "Preventing VOD in stem cell transplant patients is critically important, especially for pediatric patients who are particularly susceptible to developing VOD due to the nature of their treatment regimen. We look forward to also beginning our European trial for the prevention of VOD and transplant-associated microangiopathy in adults in the second quarter of this year.
"Earlier clinical trials of Defibrotide to treat and prevent VOD have shown encouraging results. We are pleased to begin this prevention trial in children and hope it will provide evidence of Defibrotide's potential efficacy to prevent VOD.
"We are delighted that the EBMT and the Deutsche Krebshilfe have agreed to support our efforts to develop a preventative drug for patients at risk of developing VOD, a potentially fatal disease, for which there are no currently approved therapies," concluded Dr. Ferro.
Defibrotide is a single-stranded DNA that protects the vascular endothelial cells, particularly those of small vessels, from damage and activation. After binding to endothelial cells, Defibrotide decreases cell adhesion and pro-coagulant activity of activated endothelial cells, and increases the fibrinolytic potential of endothelial cells. Defibrotide's effects are predominately local within the vascular bed, and there is no significant effect on systemic coagulation. Its beneficial pharmacological effects are due to its anti-thrombotic, anti-inflammatory and anti-ischemic properties.
Defibrotide is expected to be the subject of a U.S. Phase III study as a treatment for Severe VOD in the coming weeks. The Company also intends to initiate studies of Defibrotide to prevent VOD in the U.S. by early 2007.
VOD is a potentially life-threatening condition. The International Bone Marrow Transplant Registry estimated that approximately 45,000 people received blood and bone marrow transplants in 2002. Based on the Company's review of more than 200 published papers it is estimated that up to 20% of patients who receive blood and bone marrow transplants contract VOD. When Severe VOD occurs, blood vessels in the liver become blocked, liver failure follows and kidneys cease to function as a result of the toxic effects of cancer treatments such as high dose chemotherapy and radiation, used during SCT. Approximately 80% of patients who contract Severe VOD die within 100 days of SCT without treatment. VOD is considered one of the most important and challenging complications of SCT. There currently are no approved therapies to treat or prevent VOD.
Gentium S.p.A. is a biopharmaceutical company located in Villa Guardia (Como), Italy that is focused on the research, discovery and development of drugs derived from DNA extracted from natural sources, and drugs that are synthetic derivatives, to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate in the U.S., is an investigational drug that has been granted Orphan Drug status by the U.S. FDA to treat Severe VOD and Fast Track designation for the treatment of Severe VOD in recipients of stem cell transplants.
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