SECTION 1 : PROGRESS
THE MICHAEL J . FOX FOUNDATION 2022 IN REVIEW to develop a drug to reduce LRRK2 . Cambridge , Massachusettsbased pharmaceutical firm Biogen and San Francisco , Californiabased biotech Denali Therapeutics are advancing their promising investigation of an inhibitor of the LRRK2 gene , under the hypothesis that calming its overactivity in brain and body cells may hold significant implications for treating PD . Six hundred forty participants with early-stage PD but without a mutation in the LRRK2 gene will be recruited to test for safety and efficacy . A parallel trial is expected to launch before year-end in those who carry the LRRK2 mutation .
GBA Mutations in the GBA gene are the most common of the currently known PD genetic mutations . The GBA gene instructs production of the glucocerebrosidase ( GCase ) protein , and mutations are associated with not enough GCase activity . Canada ’ s Lawson Health Research Institute is testing a drug approved for respiratory issues , ambroxol , which may combat the mutation ’ s effects , in volunteers with PD dementia . Additionally , Indianapolis , Indiana-based pharmaceutical firm Eli Lilly continues to test a therapy to deliver a functional copy of the GBA gene to the brain , reversing some of the effects of this mutation . Both trials have the potential to stop PD progression in the LRRK2 gene ; the companies are planning a parallel trial with mutation carriers .
A Strategic Investment in the Future of Cures
Given the stratospheric cost of therapeutic development , most drug testing programs gain funding and are carried out sequentially — one step , then the next , and the next — causing practical results to take years or even decades to materialize . MJFF has long posited that deeper investment at the outset should enable simultaneous , rather than sequential , investigations , leading to faster progress and results , and increased chances of attracting follow-on investment . Now , a new MJFF funding mechanism , the Accelerated Therapies Program , is injecting select highly promising projects with greater capital in the earliest stages to enable concurrent studies and faster data collection / generation and analysis .
In February and March , the Foundation awarded about $ 5 million respectively to three biotech companies :
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