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Refanalin for lung preservation and transplantation
Phone: (516) 562-1140
DESCRIPTION (provided by applicant): Lung transplantation is the only effective treatment modality for patients with end-stage lung disease. Ischemia-reperfusion injury, associated with the retrieval, storage and transplantation of the lung is a major immu
ne-independent factor adversely affecting early graft function, graft viability and recipient morbidity and mortality. Marginal donor lungs, are even more susceptible to ischemia-reperfusion injury, and often fail transplantation. Scatter factor/hepatocyte
growth factor has significant protective activity in the setting of pulmonary ischemia-reperfusion injury and additionally, favors pulmonary epithelial repair and regeneration. However its clinical use is limited by the logistical difficulties associated
with its administration. During our Phase I program, we have identified Refanalin, an organic small molecule scatter factor/hepatocyte growth factor mimetic that improves lung function secondary to ischemia-reperfusion injury, attenuates pulmonary epitheli
al death and promotes epithelial regeneration. In a preclinical model of lung cold preservation and transplantation, Refanalin treatment reduced roentgenographic alveolar infiltration, improved pulmonary function and preserved pulmonary microarchitecture.
The present Phase II makes an in-depth evaluation of Refanalin efficacy in clinically relevant models of lung transplantation. By attenuating allograft dysfunction and preventing allograft failure, Refanalin can reduce recipient morbidity and mortality. By
attenuating ischemia- reperfusion injury in the marginal lung, Refanalin can salvage an otherwise discarded organ, and increase the donor pool. PUBLIC HEALTH RELEVANCE: A small molecule cytoprotective that can be added to the lung preservation solution an
d administered to graft recipient has significant clinical potential in lung and other solid organ transplantation.
* Information listed above is at the time of submission. *