Treating Lysosomal Storage Disorders (LSDs)

A method of screening for therapeutic agents to treat LSDs and a specific group of agents discovered thus far.


The in-vitro screening method is based on the ability of a potential agent to protect CNS precursor cells from the toxic effects of accumulating lipids in LSDs. The protection is measured by increasing cell survival rates, reducing suppression of cell division and by changes in lysosomal pH.


The screening method has identified a few therapeutic agents – both polypeptides and small molecules. These agents are expected to be used as adjunct treatments during bone marrow transplants to reduce disease severity and slow down progression or enhance the efficacy of currently used enzyme replacement therapies by restoring the low pH required for normal function of the lysosomal enzymes. These agents might also be used in other diseases with lysosomal dysfunction involvement.


The first therapeutic candidate has been reduced to practice in a murine model of Krabbe disease. In addition, several small molecules “hits” have been discovered, all of which are FDA approved drugs for other purposes.


In-vivo experiments are ongoing.

URV Reference Number: 6-2154
Patent Information:
Research Tools
For Information, Contact:
Matan Rapoport
Licensing Associate
University of Rochester
Mark Noble
Christopher Folts
Nicole Scott-Hewitt
Metabolic Syndrome