Important Research Agreement signed between Gentium S.p.A. (Gentium) and the Josep Carreras Leukaemia Research Institute.

The Josep Carreras Leukaemia Research Institute and Gentium have signed a four-year research collaboration agreement to perform further research into the beneficial effects of defibrotide for patients undergoing bone marrow transplantation.

Within the Josep Carreras Leukaemia Research Institute, researchers working on endothelial disturbance during hematopoietic stem cell transplantation (HSCT) have shown that defibrotide protects patients from some side-effects of the HSCT procedure. These studies have contributed to the recent authorization for defibrotide (Defitelio®) by the European Medicines Agency (EMA) for the treatment of severe sinusoidal obstruction syndrome (SOS) also known as Venous Occlusive Disease (VOD) of the liver. VOD is a common disorder among patients undergoing HSCT after treatment for leukaemia or other malignant blood disease.

Defibrotide has been granted orphan drug designation in the EU, U.S.A., Korea and Australia and has been made available by Gentium for over two decades on a compassionate basis and on expanded access programs. Recent studies on its effects in patients with VOD have shown that it can limit the endothelial damage that triggers the successive events that finally cause the sinusoidal obstruction. Some studies further show that defibrotide can reduce the pro-thrombotic status of these patients. However, additional research would be required to fully elucidate the precise mechanism of action of this drug in VOD and other post-HSCT complications, such as acute Graft versus Host Disease.

Through this new research collaboration agreement the Josep Carreras Leukaemia Research Institute will strengthen its research in HSCT at the Campus Clinic-UB in Barcelona. Researchers will further investigate the mechanisms involved and characterize the role of defibrotide in preventing vascular damages during stem cell transplantation.

Gentium is delighted to initiate this research collaboration with Josep Carreras Leukaemia Research Institute recognizing the wealth of knowledge and cutting-edge research carried out in the institute. As part of this collaboration Gentium will provide a significant financial contribution.

Webpage updated 02/04/2020 13:13:40