The Silverstein Foundation for Parkinson’s with GBA and The Michael J. Fox Foundation for Parkinson’

The Silverstein Foundation for Parkinson’s with GBA and The Michael J. Fox Foundation for Parkinson's Research (MJFF) held a workshop yesterday to discuss accelerating therapeutic developments for people with glucocerebrosidase (GBA)-associated Parkinson’s disease. Nearly 40 world-renowned experts from academia, industry, government and non-profits attended the meeting at the MJFF office in New York City.

Mutations in the GBA1 gene are present in up to ten percent of people with Parkinson’s disease (PD), representing one of the most common genetic risk factors for this disorder. GBA1 encodes for glucocerebrosidase (GCase), a lysosomal enzyme with decreased activity in both genetic and sporadic cases of PD.

Over the last few years, understanding of GBA-associated Parkinson’s disease has grown substantially, and in 2017, clinical trials began testing a therapy in GBA1mutation carriers for the first time. In the workshop, The Silverstein Foundation and MJFF sought to continue building momentum around GBA biology and therapeutic development for the benefit of people with genetic and sporadic PD.

"GBA has a genetic connection to Parkinson’s disease but its dysfunction is also found in some people with no known cause of the disease," said Brian Fiske, Ph.D., MJFF Senior Vice President of Research Programs. "This makes this area of research a high priority with great potential impact for the broader Parkinson’s population."

By jointly reviewing the state of the field, the Foundations sought to identify key studies and initiatives that will facilitate and advance therapeutic development. MJFF and The Silverstein Foundation also are partnering on a research funding program to be announced later this month.

"We are excited to be joining forces with the MJFF to support research that will lead to new therapeutics in the field," said Jonathan Silverstein.

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