Since 2000, The Michael J. Fox Foundation has funded more than $450 million to speed a cure for Parkinson's disease.
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Case Studies — Progress for Parkinson's Patients
Translating Genetic Findings into Real Treatments
Insights from Parkinson's genetics studies over the past 15 years have revolutionized the search for a cure. One of the most important genetic targets in Parkinson's, LRRK2, was discovered in 2004. With over $57 million in investments to date, MJFF's outside-the-box approach to LRRK2 drug development aims to make LRRK2 as relevant in the doctor's office as it is in the research journal.
Backing a Parkinson's "Vaccine"
In 2012, Austrian biotech and MJFF awardee AFFiRiS AG secured major investment in the first vaccine approach to Parkinson's. By stimulating the body's immune system to produce antibodies against a protein that clumps in the body cells of people with Parkinson's, AFFiRiS aims to clear these clumps, slowing disease progression. With $1.5 million in support from MJFF, the company is now in the first stage of clinical testing.
Catalyzing New Treatments Based on Enzyme Activity
Lowered activity of a certain brain enzyme is associated with alpha-synuclein accumulation, the hallmark pathology of Parkinson's. Biotech Amicus Therapeutics and pharmaceutical company Biogen Idec are partnering to investigate whether increasing the activity of this enzyme could help prevent or slow progression of disease. The work could lead to a new class of small molecules for the treatment of Parkinson's disease. Initial funding from MJFF allowed Amicus to gather the data needed to secure the partnership with Biogen.
Visualizing Alpha-Synuclein in the Living Brain
The alpha-synuclein protein forms clumps in the body cells of all people with PD. These clumps, seen at autopsy, remain the only definitive diagnosis of Parkinson's disease. The Foundation is spearheading a $1.5-million research consortium to develop a sophisticated brain imaging technique that would reveal alpha-synuclein's distribution in the living brain, dramatically impacting our ability to diagnose Parkinson's and measure the effectiveness of drugs targeting its progression.
Sponsoring a Landmark Biomarkers Study
The Parkinson's Progression Markers Initiative (PPMI) aims to forge a critical missing link in our pursuit of a cure. Launched in 2010 and sponsored by MJFF, the $55-million project has won the enthusiastic buy-in of academic and industry researchers, government and pharmaceutical partners, and hundreds of patients and families. An effort of this magnitude required MJFF to chart an entirely new course, taking on an unprecedented role for a private research funder. Today the study is not only bearing fruit but has become a new model for collaborative research. Learn More
Looking Beyond Dopamine
In 1998, MJFF awardee Jeff Conn of Vanderbilt University identified a glutamate receptor called mGluR4. In 2007 we awarded his team over $4 million to show that mGluR4, which bypasses the dopamine system altogether, can lead to functional improvement in Parkinson's symptoms. In 2012, based on Conn's work, pharmaceutical giant Bristol-Myers Squibb signed on with Vanderbilt to develop drugs targeting mGluR4. Learn More
Improving Therapeutic Consistency
Current medication for Parkinson's is taken orally, which can slow and result in inconsistent absorption, leaving patients waiting for it to take effect and not knowing how long relief will last. Beginning in 2011, MJFF funded clinical testing of CVT-301, an inhaled formulation of levodopa being developed for rapid and reliable relief from debilitating motor fluctuations (OFF episodes) associated with PD. The developers, Civitas Therapeutics, Inc., announced that venture capital firms have committed them $38 million, providing for late-stage development of the therapy.
Helping Dyskinesia Researchers Row in the Same Direction
In 2011/12, MJFF sponsored a $1-million study to identify the best clinical scale for detecting dyskinesia, the uncontrollable movements that are a side effect of Parkinson's treatment. Up to that point researchers had been using many different scales, making it difficult to establish consensus in the field or compare results across trials. Having a single, validated scale allows for better design of conclusive clinical trials.
Crowd-sourcing Better Research Tools
We create our own, IP-free experimental tools (such as laboratory reagents) and share them free of charge with research teams in exchange for feedback on how well they work. Then we make the highest-performing tools available at low cost through the MJFF catalog. It's a common-sense approach leveraging the wisdom of the crowd to keep researchers focused on new visions for curing Parkinson's disease.
Assembling a Standing Army of Clinical Trial Volunteers
Clinical trials are the final, essential stage in developing any new drug. Across all diseases, 85 percent of trials face delays and 30 percent never get off the ground because of difficulty finding volunteers. MJFF launched Fox Trial Finder in 2012 to make it easier for thousands of patients and loved ones to step up for trials and keep experimental Parkinson's treatments moving toward pharmacy shelves. Learn More
Dismantling Business and Regulatory Hurdles in Drug Development
MJFF takes a problem-solving approach to business and regulatory hurdles that slow the development of new treatments. For example, in 2013, MJFF is engaging the U.S. Food and Drug Administration (FDA) in efforts to define a roadmap for bringing Parkinson's cognition treatments to market. Learn More