Principal Investigator:    Andrew Nichols
Grant Title:    Evaluation of CAT-4001 in Frataxin-deficient mouse models and DRG neurons to enable its therapeutic development to treat Friedreich's Ataxia
Status:    Ongoing
Start Date:    12/15/2015
Duration:    24 months
Description:    In Friedreich’s Ataxia (FRDA), the deficiency of frataxin expression leads to oxidative stress that may play a significant role in disease pathology. Impaired nuclear translocation of Nrf2, a transcription factor which regulates cellular responses to oxidative stress, may be the causative factor for the oxidative neuronal damage in FRDA. In addition, cardiac and neuronal inflammation may play a role in the pathology of FRDA. Thus, activation of Nrf2 to drive antioxidant pathways and inhibition of NF-kB to reduce inflammation may represent a good approach to the treatment of FRDA. We therefore propose to test the functional activity of CAT-4001, a novel small molecule designed to activate Nrf2 and simultaneously inhibit NF-kB, in animal and cellular models of FRDA