The Silverstein Foundation for Parkinson’s with GBA and The Michael J. Fox Foundation for Parkinson’s Research (MJFF) Jointly Announce Availability of $3.5 Million in Fall 2018 Research Funding to Expand Understanding of the GBA gene in Parkinson’s Disease

New York (March 19, 2018) – The Silverstein Foundation for Parkinson’s with GBA and The Michael J. Fox Foundation for Parkinson’s Research (MJFF) today announced the availability of $3.5 million in Fall 2018 research funding to expand the understanding of the GBA gene in Parkinson’s Disease and to accelerate novel therapeutic development.

Mutations in the glucocerebrosidase (GBA1) gene are one of the most common risk factors for Parkinson’s disease. GBA1 encodes a lysosomal enzyme, b-glucocerebrosidase (GCase), which is deficient in Gaucher’s disease (GD). People with GBA1 mutations have an increased prevalence of PD.  While mutations in the GBA gene account for five to 10 percent of all Parkinson’s cases (making it one of the most common genetic contributors to PD), GBA protein dysfunction has been observed even in Parkinson’s patients who do not carry mutations in the GBA gene. This potential broad base of benefit makes it an especially compelling drug target. This program seeks to increase understanding of the role of GBA in PD disease processes and to accelerate and facilitate the delivery of drugs targeting GBA mutations in people with Parkinson’s.

The program announced today aims to advance understanding of the pathophysiological cellular mechanisms triggered by GBA1 mutations, as well as the mechanisms that trigger GCase dysfunction in the absence of mutations. In addition, the Foundations aim to promote the development and testing of novel therapeutic approaches that can prevent the pathogenic mechanisms triggered by GBA1 mutations.

Applicants may propose studies that focus on:

  • Biological Pathways and Novel Targets
  • Therapeutic Development

The Foundations will prioritize proposals utilizing experimental approaches, such as patient-derived material and/or animal models utilizing clinically translatable endpoints.

Applicants may request up to $150,000 for biological investigations or $500,000 for novel therapeutic development.  Further details and registration for the information webinar may be found on The Silverstein Foundation’s website under Request for Applications on the homepage.