Parkinson’s Disease (PD) is a progressive neurological disorder whose prevalence increases with age and certain genetic predispositions.  It is estimated that 7-10 million people are affected by Parkinson’s Disease worldwide, with 1 million in the United States.  Currently, there are no small molecule drugs to halt or reverse the progression of PD.  The gold standard therapy for treating the symptoms of PD is dopamine replacement therapy with Levodopa (L-dopa).  However, 60-80% of patients treated with L-dopa develop complications, including a severe movement disorder called L-dopa-induced dyskinesia.

To address the unmet need of pharmaceutical interventions that can halt the progression of Parkinson’s Disease, researchers at Drexel University have developed GT951, a novel small molecule activator of glutamate transporter GLT-1 / excitatory amino acid transporter EAAT2.  GT951 confers neuroprotection by scavenging out excess glutamate to prevent excitotoxicity and subsequent neurodegeneration.  In preliminary studies, GT951 has demonstrated high specificity to GLT-1 and sub-nanomolar efficacy in removing glutamate from the synapse.  A pilot efficacy study in a rodent PD model demonstrated that GL951 can prolong the time taken to form lesions and significantly reduce disease severity.  GT951 is being optimized for pharmacokinetics, safety, and toxicology to develop a preclinical candidate for GLP studies.