LRRK2 Summit

On 9 December 2009, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will be hosting a meeting to discuss the Parkinson’s disease (PD)-associated factor leucine-rich repeat kinase 2 (LRRK2).  Mutations in LRRK2 represent one of the most common genetic links to PD discovered to date—an estimated 1-2% of cases—and given its putative role as a protein kinase, LRRK2 has garnered much excitement among drug developers as a possible novel therapeutic. Accordingly, MJFF has made it a priority to promote and facilitate further study and translation of LRRK2. To date, our efforts have focused on initiatives to understand LRRK2 biology, develop relevant tools and assays, increase clinical and genetic understanding and promote therapeutic development, all with the hope of accelerating new treatments for PD.

The goal of this meeting is to assess the current state of the field, examine what activity is currently ongoing (including MJFF efforts), determine what the remaining challenges are, and ultimately recommend ways MJFF and the field can prioritize and advance future LRRK2 efforts. Key topics will focus on biological and clinical/genetic understanding, tools and resources, and therapeutic development.

Each session at the meeting will include a presentation on the current status of the field, a presentation from MJFF staff on areas we are supporting and finally a discussion about where the next 1-2 years of work should focus.

Below is the essential breakdown of discussion topics to be covered at this meeting as well as the primary questions each session is attempting to address:

Biological Understanding of LRRK2  --  Current Status and Future Directions

• What are the most critical unknowns regarding LRRK2 normal and pathological function?

Research Tools and Reagents

• What critical tools/reagents would significantly accelerate research around LRRK2 understanding?

LRRK2 PD: Genetics and Clinical Phenotype -- Current Understanding and Future Directions

• What are the most critical unknowns regarding LRRK2 genetic/clinical understanding?

LRRK2 as a Therapeutic Target – Industry Perspective

• What information does Industry need to most effectively develop and successfully test potential LRRK2 therapeutics?
• What would the first LRRK2 efficacy clinical trial look like?

You can start the discussion on these questions now, and fill us in on points you think we've left out of the agenda. Just log in and click the orange 'create response' button.