Study Rationale: Genetic mutations resulting in a depletion of glucosylceramidase activity is the most predisposing risk factor for developing Parkinson’s disease (PD). Activating glucosylceramidase to slow down or reverse symptoms has been proposed as a promising therapeutic opportunity for many years. BXQ-350 is a novel biologic that contains the protein that naturally activates glucosylceramidase activity. BXQ-350 is currently developed as an anticancer agent and results indicate that it significantly stimulates glucosylceramidase activity.
Hypothesis: We hypothesize that the effects of BXQ-350 that have been observed in cancer models translate into PD models and would justify initiating a clinical study in people with PD.
Study Design: The proposed studies will determine the efficacy profile of BXQ-350 in multiple preclinical PD models and assess the best way to administer BXQ-350 to people with PD. These results will be critical to initiate a clinical study and will be presented to FDA for input on how the first human clinical study should be designed and performed.
Impact on Treatment of Parkinson’s disease: Activating glucosylceramidase should not only improve symptoms of PD, but also delay disease progression. Thus, BXQ-350 could provide significant benefits to people with PD, including better quality of life and longer survival.
Next Steps for Development: Results of this proposal will determine whether BXQ-350 activates glucosylceramidase in preclinical PD models and if a clinical study is warranted. Next steps would include submitting an IND to FDA and initiating a Phase 1 study in people with PD.