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Preclinical Optimization of a Novel Neuroprotective Agent to Treat Parkinson’s Disease

Project Team

  • Sandhya Kortagere, PhD, Microbiology
  • Jill Farmer, DO, MPH, Neurology/ Director of the Parkinson's Disease and Movement Disorder program at DUCOM


Huntington’s Disease (HD) is a fatally inherited neurodegenerative, orphan disorder affecting 1 in 10,000 individuals with a higher prevalence in Caucasian race. HD is caused by an expanded glutamine repeat in the N-terminal region of the huntingtin (HTT) protein. The disease onset has distinct patterns in juvenile and adult patients, but with similar disease progression leading to death within 10-20 years of onset. The symptoms of HD are very similar to that of Parkinson’s Disease (PD) with motor and cognitive impairment and choreic (dyskinesia) involuntary movements that significantly increase with disease progression.

Currently there are no drugs to treat or halt the progression of HD. A few antipsychotic drugs and other medications have shown efficacy in treating the symptoms of the disease in some patients. Dysregulation of glutamate in the corticostriatal network is believed to play a major role in the pathology of HD. High expression of the mutant HTT is significantly correlated with the downregulation of the astrocytic glutamate transporter -EAAT2 responsible for 90% of the glutamate uptake in the brain. Thus, to address the significant unmet need we have developed GT951, a novel small molecule activator of EAAT2 that has shown significant promise in disease modifying features of Parkinson’s disease and is anticipated to work similarly in HD. In this study, we propose to test GT951 and its analogs first in a human HTT mutant induced drosophila HD model and advance one best compound to test in a rodent model of HD.