Abstract
Since 2005, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has invested significant funding and non-funding effort to accelerate research and drug development activity around the Parkinson disease (PD)-associated protein LRRK2. MJFF has spearheaded multiple public/private pre-competitive collaborations that have contributed to our understanding of LRRK2 function; de-risked potential safety questions around the therapeutic use of LRRK2 kinase inhibitors; and generated critical research tools, biosamples, and data for the field. Several LRRK2-targeted therapies are now in human testing due to the hard work of so many in the PD community. In this perspective, we present a holistic description and model of how our Foundation’s support targeted important barriers to LRRK2 research and helped move the field into clinical trials.
Highlights
Our efforts have focused on a multi-faceted strategy with the goal of informing optimal clinical trials of leucine-rich repeat kinase 2 (LRRK2)-targeted therapies
Using a similar collaborative approach, MJFF funded a global team of scientists and drug makers who had access to critical tools and model systems to identify and validate major downstream substrates of LRRK2
MJFF support aims to define the upstream modifiers of LRRK2 activation and downstream mechanisms of LRRK2-mediated neurodegeneration using novel methods and model systems
Summary
The nomination of genes, proteins, and pathways potentially involved in PD pathogenesis drives. In the few years, we hope to gain even more detailed structural information on the protein along with further information about the intra- and inter-molecular interactions that regulate LRRK2 function This will pave the way for refinement of current kinase inhibition strategies and development of new strategies for targeting LRRK2. Using a similar collaborative approach, MJFF funded a global team of scientists and drug makers who had access to critical tools and model systems to identify and validate major downstream substrates of LRRK2. MJFF support aims to define the upstream modifiers of LRRK2 activation and downstream mechanisms of LRRK2-mediated neurodegeneration using novel methods and model systems These studies are yielding interesting data on other proteins and pathways that could be targeted to decrease the pathogenic effects of LRRK2 [20,21]. LRRK2 therapies may benefit a larger portion of the PD population
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