Study Rationale & Design:
Components of blood have been found to improve health and enhance multiple biological functions in pre-clinical studies of age-related decline. Blood plasma has been particularly important in regulating the complex mechanisms that can cause these effects. The efficacy of plasma is being studied in the clinic for multiple indications. Alkahest has identified a proprietary plasma protein fraction, GRF6019, which shows positive effects on cognitive and motor function in old models. Plasma and plasma protein fractions have been used as human therapies for decades. Some advantages of GRF6019 over plasma may include increased safety, convenience and treatment benefits. Based on the effects observed in laboratory studies, Alkahest is conducting a Phase II study to explore the safety and efficacy of GRF6019 in people with Parkinson's disease (PD).
Impact on Diagnosis/Treatment of Parkinson's Disease:
GRF6019 is a plasma protein-based therapy with several possible mechanisms of action. Study results will shed light on the safety and therapeutic effects of GRF6019 in people with PD. The results will also help identify effective doses and endpoints for future studies. During this study, patient data will be collected and analyzed to determine any relationships to disease progression or observed treatment benefits.
Next Steps for Development:
After this study, we will have a more complete understanding of the safety and efficacy of GRF6019 in people with PD. The results will enable us to focus on the endpoints that are most impacted and select participants most likely to benefit from this therapy in future studies. Alkahest will also measure changes in participant plasma that may be related to therapeutic effects.