Use of Acid Ceramidase For The Treatment of Lung Disease


Cystic Fibrosis (“CF”) is the most common autosomal recessive disorder in Europe and the USA, impacting one in every 2500 children born in western countries. It is a genetic disease caused by mutations of the CF transmembrane conductance regulator protein (“CFTR”).  While this genetic mutation leads to several respiratory, reproductive and gastrointestinal complications, the primary cause of morbidity and mortality in these subjects results from the destructive effects of chronic pulmonary colonization with Pseudomonas aeruginosa (“P. aeruginosa”).


P. aeruginosa infection of epithelial cells is initiated by contact of the pathogen with the cell surface. Therefore, identification of the molecular factors that makes CF patients highly susceptible to infection by P. aeruginosa, is critical to the development of new strategies for the treatment and prevention of CF. Dr. Edward Schuchman and collaborators at the University of Duisburg-Essen and Weizmann Institute have recently shown that the lipid ceramide is elevated in the bronchial epithelial cells of the CF patients, leading to enhanced binding of P. aeruginosa to CF respiratory epithelial cells.  Dr. Schuchman and his colleagues also showed that administering an inhaled version of the enzyme, acid ceramidase (AC), which hydrolyzes ceramide in CF mouse models, prevented infection of P. aeruginosa.  Therefore, AC can be used in the treatment of CF to enhance existing therapies and to prevent infection by this and perhaps other pathogens.


Patent Status: Patent Pending


Technology: Method

Patent Information:
For Information, Contact:
Jeanne Farrell
Business Development Director
Mount Sinai School of Medicine
Edward Schuchman
Erich Gulbins
Anthony Futerman
Yael Pewzner-Jung
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