MJFF Brokers Industry Consortium to Advance Disease-modifying Therapies

FoxFeed Blog

Posted by  Maggie McGuire, February 04, 2015

In our pursuit of a cure for Parkinson’s disease, we’re tenacious, resilient, problem solvers. Our unique view of the research landscape and our unbiased dedication to meeting patients’ needs allows the Foundation to lead initiatives that advance progress across the board.

For example, the Parkinson’s Progression Markers Initiative is working toward validated biological markers of PD that will accelerate drug testing, and Fox Trial Finder is connecting research volunteers with clinical studies that desperately need them to evaluate new therapies.

One of the latest on our list of MJFF-led strategies and projects is the LRRK2 Safety Initiative. The Foundation has united three powerhouse pharmaceutical companies for a pre-competitive consortium to characterize the effects of LRRK2 inhibitors and advance these potential disease-modifying medications.

Studies Find Potential Hurdle to Drug DevelopmentMutations in the LRRK2 gene are associated with Parkinson’s disease and cause heightened activity of the LRRK2 protein kinase, which modifies other proteins. Researchers hypothesize that lowering that activity with drug inhibitors could protect brain cells. 

In a paper published today in Science Translational Medicine authors from biotechnology company Genentech and from MJFF share that they observed changes in lung tissue of pre-clinical models after introducing a LRRK2 inhibitor.

“We don’t know if the changes would have a negative effect, but really any modification is a reason to pause and to consider the safety of the therapy for humans,” said Brian Fiske, PhD, MJFF vice president of research programs and an author on the paper.

MJFF Organizes Drug Development Leaders to Move ForwardHowever, we’re not stepping back from LRRK2. This protein remains one of our top priority targets for a therapy to stop or slow Parkinson’s. 

In light of the concerns around LRRK2 inhibitors, MJFF held a meeting with leaders in this field in January 2014. Kinase inhibitors are a familiar class of drugs to pharmaceutical companies, so a handful had compounds against LRRK2 in their pipelines.

Attendees strategized the future of drug development in this area, and key players in the industry — Genentech, Merck and Pfizer — signed on to join the MJFF LRRK2 Safety Initiative. Each member has submitted a different LRRK2 inhibitor compound to an MJFF-chosen neutral site for independent testing, and data is shared across the consortium. By working together, they’re more efficiently growing the field-wide understanding of the safety and impact of LRRK2 inhibitors.

“Research is a business, and it’s unfortunately often competitive,” said MJFF CEO Todd Sherer, PhD. “These companies are truly putting patients first in their collaboration to move drug development projects forward to clinical testing and application for the five million living with Parkinson’s disease.”

“We can learn more and move faster by working together,” said Reina Fuji, VMD, PhD, senior scientist-pathologist at Genentech. “The Michael J. Fox Foundation’s brokerage of this consortium assures its integrity and patient focus. We at Genentech are glad to contribute to research in this area as a part of the LRRK2 Safety Initiative.”