Study Rationale & Design:
Components of blood have been found to improve health and enhance multiple biological functions, including brain function, in older animals. Blood plasma has been particularly important in regulating the complex mechanisms that can cause these effects. The beneficial effects of plasma components called proteins is being studied in multiple human diseases associated with aging. Alkahest has identified a proprietary plasma protein fraction, GRF6021, that shows positive effects on cognitive and motor function in models of Parkinson’s disease. Some advantages of GRF6021 over whole plasma include increased safety, convenience and potentially better treatment benefits. Based on the effects observed in model studies, Alkahest is conducting a Phase II study to explore the safety and efficacy of GRF6021 in people with Parkinson’s disease (PD) and mild cognitive impairment or dementia. Alkahest is also conducting a similar study with another selected plasma protein fraction in Alzheimer’s disease.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
GRF6021 is a plasma protein-based therapy with several possible mechanisms of action. Study results will help us understand the safety and tolerability of GRF6021 infusions in individuals with PD, as well as determine whether there are any beneficial effects. The results of this study will be used to conduct a larger, more definitive study in the future, so that ultimately all patients with PD and cognitive impairment may benefit.
Next Steps for Development:
After this study, we will have a more complete understanding of the safety and efficacy of GRF6021 in people with PD. The results will enable us to focus on the specific cognitive domains that are most impacted and select the patients who are most likely to benefit from this therapy in future studies.