CCTST Just In Time (JIT) Awardee Highlight

CCTST Just In Time (JIT) Awardee Highlight

In 2017 Cincinnati Children’s Hospital investigator, Dr. Manoj Pandey, PhD., was awarded Center for Clinical and Translational Science Just In Time (CCTST-JIT) funding for the project, “C5a-C5aR1 function for brain storage of glucosylceramide in Gaucher disease”.  The goal of this study was to investigate if the protein complement 5a (C5a) interaction with its receptor (C5aR1) triggers the excess brain accumulation of the fatty molecule, glucosylceramide (GC), which sparks inflammation and can lead to neurodegeneration in Gaucher Disease. 

Gaucher disease is a rare disease, with an incidence of about 1 in 60,000 in the general population. Worldwide there are about 4,000 Gaucher disease patients. Certain therapies, such as enzyme replacement therapy and substrate reduction therapy, treat many of the visceral aspects of Gaucher disease but demonstrate diminished or no response in the brain and for which there is not an effective therapy. The results obtained from the JIT funded study provide new insights that might prove useful to design novel adjunctive therapeutic approaches that may apply to manage disease complications in patients with Gaucher’s, Parkinson’s and other lysosomal storage diseases.

This JIT project has led to two publications, funding from the Michael J. Fox Foundation, and Media Coverage:

The mission of the CCTST-JIT grant mechanism is to enable investigators to use University of Cincinnati or Cincinnati Children’s Hospital Medical Center Core facilities to obtain critical data for submission of a competitive extramural proposal, patent application or commercialization agreement.  The JIT application cycle is open from September 9, 2019 - October 7 2019; click here for details.

 

Post Date: September 6, 2019