We are actively reviewing pre-clinical programs that could be appropriate for GBA mutation carriers.
The Silverstein Foundation has funded the following research:
- A grant was made to the Michael J. Fox Foundation for Parkinson’s Research which will be directed specifically to GBA-related research. (March, 2017)
- A grant was made to the Parkinson’s Institute and Clinical Center in San Francisco to allow Executive Director, Dr. Carrolee Barlow and her team to complete a study of the clinical manifestations of genetic (GBA, LRRK2, etc.) as compared to sporadic Parkinsons, specifically as it relates to neurons in the GI tract. Studying people who have a genetic form of Parkinson’s will allow scientists to determine if the same mechanism that is believed to cause sporadic Parkinson’s (which includes ~90% of people with the disease) also causes Parkinson’s in people with GBA mutations. It is widely believed that targeting alpha-synuclein (the suspect in sporadic Parkinson’s) will also be helpful in people with GBA-related Parkinson’s. However, it remains to be proven that this is the case. There are several potential therapies that might be useful for stopping Parkinson’s in people with GBA and other genetic mutations that do not directly target alpha-synuclein. Data from this study can be used by pharmaceutical companies in collaboration with Dr. Barlow’s team and the FDA to design and quickly advance pivotal clinical studies with disease-modifying therapies for people with GBA mutations. (March 2017)
- A grant was made to a private biotechnology company developing novel therapeutics in GBA PD and other neurodegenerative diseases.
- A grant was made to Columbia University which will be directed specifically to GBA-related research.
- A grant was made to Columbia University which will be directed specifically to GBA-focused bioinformatics.