This article originally ran in our Fall 2013 newsletter The Fox Focus on Parkinson’s.
Virtue or not, patience is not something you find a lot of at The Michael J. Fox Foundation. There is still much to be done to find the cure for Parkinson’s disease, and we’re working urgently to get there.
MJFF speeds the delivery of new therapies by identifying research roadblocks and building the infrastructure to overcome these challenges. While one might think of the lab scientist scratching his head in front of a row of test tubes, the race toward discovery includes non-scientific hurdles, too. Difficulties in recruiting clinical research volunteers, sharing resources and obtaining regulatory approval can all slow the already long journey from hypothesis to clinically available treatment. Foundation initiatives are working to move past these impediments to breakthroughs.
Connecting patients and healthy volunteers with studies
Before a new drug or treatment can be made available to the public, scientists must prove that the intervention is safe and effective and figure out what dose works best. Clinical research needs volunteers to help answer these questions, but studies have strict requirements for participation.
It can be difficult to find people who meet these eligibility requirements. It is even more difficult to find people who are eligible, live near the study site and want to participate. The frustration works both ways, too. As an enthusiastic potential research volunteer, you may run into trouble finding the studies in your area that you are eligible for. Some scary statistics: Thirty percent of all clinical trials fail to recruit a single subject, and 85 percent of trials finish late due to recruitment troubles. Despite a willingness to participate, fewer than 10 percent of PD patients take part in trials.
The MJFF Fox Trial Finder Web site lets interested Parkinson’s patients and their loved ones (who can participate as healthy controls) create a secure profile with basic demographic information and medical history. The tool reviews clinical studies in our database and refers matches based on geography and eligibility requirements. Fox Trial Finder also lets researchers search the database for volunteers who may be eligible for their studies. With more than 23,000 volunteer profiles and over 300 studies at sites around the world, the system advances research by bridging the gap between interested participants and the studies that need them.
Sharing resources to identify biomarkers
Most studies exist in silos where researchers recruit their own volunteers, collect and catalogue data as they see fit, analyze their results and then publish their findings years later. There is a better way. The MJFF-led Parkinson’s Progression Markers Initiative, which aims to identify biological markers of PD onset and progression, makes de-identified data from its more than 660 participants available to qualified researchers after an easy online request process. To date, PPMI data has been downloaded close to 90,000 times by researchers around the world, and the initiative has received 27 requests for biosamples.
As PPMI grows this year to now examine pre-motor symptom biomarkers of PD, investigators will continue to share data. This real-time, open-access model strategically hastens the pace of research toward drug development and a cure.
PPMI also sets the stage for a quicker next generation of research. When drug compounds are ready for clinical testing, investigators have a pool of volunteers from PPMI who have already shown interest in participating in research and for whom researchers have a rich profile of information. Additionally, if PPMI characterizes a reliable biomarker for PD, drugs can be tested faster. Rather than waiting for a change in clinical symptoms, researchers can measure any change on a molecular level. That information on the drug’s results affects advancement through clinical testing and regulatory approval.
Addressing regulatory issues
MJFF is taking other steps to move drugs through the “valley of death” from laboratory discoveries to medications in patients’ hands. Foundation researchers co-chaired a meeting with 40 key leaders, including 16 representatives from the U.S. Food and Drug Administration, in April to identify the regulatory requirements for pursuing therapy for mild cognitive impairment with Parkinson’s disease (PD-MCI).
Better understanding of who PD-MCI patients are, how to recognize PD-MCI through diagnosis, and how to measure improvement in these patients are critical steps to filling an unmet need so that treatments can be tested in the clinic and ultimately approved by regulatory agencies,” wrote MJFF’s Lona Vincent, MPH, and Jamie Eberling, PhD, in an outcomes summary.
Attendees of the regulatory roundtable deemed that existing cognitive scales can be utilized for trials in the short-term, but to better serve patients’ needs, the PD field can develop its own optimal scale for later studies. By driving these discussions, the Foundation progresses research down the road to clinically available therapies.
At MJFF we’re in the business of answers. We strive to stay informed about the process of making things happen, and we’re taking action. We’re identifying the problems along the way and building infrastructure to overcome them to move us toward the big answer patients need: the cure for Parkinson’s.
Taking Risks to Reap The Rewards
The drug development industry is a numbers game. To invest in a potential therapeutic, pharmaceutical companies, and even the government, ask researchers to show them why they think the intervention will work, and they want data. Researchers often face a funding dilemma — they design studies to get to more data…but, how do they get their studies off the ground before the compelling data exists? Parkinson’s researchers come to The Michael J. Fox Foundation. We’re strategic and ask for basis and background of a concept, too, but we take chances. We’re eager to act on ideas faster, and we’re not satisfied with the shuffle of drug development. We want to leap ahead.
MJFF invests in high-risk, high-reward projects to accelerate progress. Initial findings make these studies more attractive to industry and government, and we’re happy to hand them off. That means we can focus on the next wave of innovation, and we’ll keep it up until we get to a cure.