Pushing forward collaborations in the GBA1 field

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Although GBA1 mutations are much more common in Parkinson’s disease than LRRK2 mutations, the research on GBA1 is lagging in terms of dedicated funding and scale.

Recently, thanks to a large grant from The Hilary and Galen Weston Foundation, the Van Berkom Family, and other funders, we have established G-Can, the GBA1 Canada initiative. The main goal of this initiative is to create a pre-competitive, open science platform to advance GBA1 research and treatment. This initiative will generate tools and data to support GBA1 basic and clinical research, support the creation of trial-ready populations for GBA1 clinical trials, and provide funding for GBA1-dedicated research. 

At least 25 companies and foundations sent their representatives to the meeting. With talks from prominent clinicians and researchers, the presence and presentations by many early-career researchers, as well as the presence of patient and caregiver representatives, this meeting created many networking opportunities, interesting discussions, and future collaborations. Three awards were given for the best research presented by a trainee; the winners were: 1st prize: Maxime Teixeira; 2nd prize: Emma Somerville; and 3rd prize: Ruth De-Paula.    

There were numerous important takes from this meeting, yet we can only briefly mention several of them here. In basic science research, multiple talks have emphasized understudied areas of GBA1 research, which is usually focused on the lysosome. These included effects on the immune system, mitochondria, and lipidomics within and outside of the lysosome. These areas and others should not be neglected, as they might provide novel insight on how GBA1 is involved in Parkinson’s disease. Other very important discoveries reported in the meeting were made by genome-wide CRISPR inhibition and activation screenings that identified novel potential modifiers of GBA1, which in the future can serve as therapeutic targets. 

Among many other interesting presentations, Dr. Oluwadamilola Ojo from Nigeria gave a fascinating talk about the discovery of the novel African variant in GBA1, found in about 40% of African individuals with Parkinson’s disease, as well as on challenges that clinicians and researchers in Africa are facing. The discovery of this very common variant makes the African population an excellent target for clinical studies and clinical trials, therefore tackling these challenges will be crucial.  

On the clinical side, there were very interesting talks and discussions that covered all translational efforts and ongoing clinical trials. Dr. Pablo Sardi gave an inspiring, honest talk about lessons to learn from the failure of the MOVES-PD trial. Many other therapeutic approaches, from small molecules to gene therapy, are being explored and are in different phases of development and were discussed in depth, including discussions on the potential ambroxol for treatment of both Parkinson’s disease and Gaucher disease. 

Our plan is that the GBA1 meeting will become an annual international meeting that will continue bringing together all stakeholders involved in GBA1 research – researchers, clinicians, foundations, industry, and of course, patients and caregivers. Looking forward to seeing everyone at the 2025 meeting!  

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