8 Transforming the Drug Development Landscape
LRRK2 : Enabling Progress through the Pipeline
Mutations in the LRRK2 gene cause a small minority of Parkinson ’ s cases , but the biological changes found in these individuals yield critical clues for the development of better Parkinson ’ s treatments that would benefit everyone with the disease — genetic mutation or not .
In 2020 , two major players in Parkinson ’ s drug development — San Francisco biotech Denali and Cambridge pharmaceutical firm Biogen — announced the launch of a late-stage trial of their therapy targeting the LRRK2 protein . As this report goes to press , the highly anticipated results from this trial are expected by year-end 2021 . Additionally , Biogen is testing a separate approach targeting this same high-priority pathway .
LRRK2 was discovered in 2004 and quickly emerged as one of The Michael J . Fox Foundation ’ s most important areas of focus . ( One of the first major studies of LRRK2 was funded at the pharmaceutical firm Wyeth through our Therapeutics Development Initiative program in 2006 .) Today , as LRRK2 continues its steady march toward market , it is fair to say that the entire field as it exists today could only have been achieved through key findings and interventions conceived and financially enabled by MJFF . ( See “ Tapping into a Promising LRRK2 Pipeline ,” page 10 .)