Study Rationale: Our project will test the effectiveness of a new treatment for Parkinson’s disease (PD). Developed by Sanofi, the drug — called Tolebrutinib — blocks inflammation in the brain. Based on our studies to date, we believe that Tolebrutinib, which reaches high concentrations in the brain, could block several of the mechanisms that contribute to the onset and progression of PD, including persistent inflammation, mitochondrial dysfunction, infiltration of harmful inflammatory immune cells and the death of dopamine-producing neurons.
Hypothesis: We propose that Tolebrutinib could be a safe and effective treatment for PD based on our results in preclinical animal models and our ongoing clinical trials with this drug for other diseases.
Study Design: We will test the effectiveness of Tolebrutinib in cell culture and in multiple preclinical animal models of PD. We will determine whether Tolebrutinib can prevent or rescue PD pathology in these models, which would indicate that this drug could have potential to be tested as a therapeutic in people with PD.
Impact on Diagnosis/Treatment of Parkinson’s disease: There are currently no treatments for PD. Our drug, Tolebrutinib, has been shown to be safe and well-tolerated in clinical trials for multiple sclerosis. If this project is successful, Tolebrutinib can therefore directly enter clinical trials as a new treatment for PD.
Next Steps for Development: If our testing in preclinical models produce positive results, we will be able to progress this drug into Phase 2 clinical trials for PD to determine if it could be a new means by which to slow or halt disease progression.