“The road to a cure is not a straight line.”
Our Foundation exists to pursue a single, exclusive goal: accelerating a cure for Parkinson’s disease (PD). But the road to a cure is not a straight line. It is lined with way stations, unexpected detours and countless make-or-break moments.
One example is the leap from laboratory testing to clinical trials in humans. Though thousands of drugs are tested at the lab bench, only a tiny percentage prove safe and effective enough to warrant testing in people. Advancing to clinical trials is a true research milestone.
The Michael J. Fox Foundation (MJFF) pushes promising drugs toward that threshold by investing in the most promising early-stage treatments. We seek out studies with great scientific rationale but in need of resources to test that rationale.
It’s a high-risk, high-reward approach. And it is paying off.
This year has seen enormous progress in the pursuit of a treatment to slow or stop Parkinson’s progression, with several companies launching trials and multiple projects already in human testing moving closer to FDA approval.
In fact, four companies are now in human testing of therapies against the most important Parkinson’s drug target, alpha-synuclein. (Similar to beta-amyloid in Alzheimer’s, alpha-synuclein clumps in the brain and body cells of everyone with Parkinson’s disease. Researchers believe that preventing or clearing these clumps could slow or stop PD.) MJFF funded two of these companies in early phases of their investigations, and we’re partnering with all four to keep their studies moving forward toward practical treatments for patients.
Other targets, including LRRK2 and GBA, also are advancing through the PD drug pipeline. MJFF is leading research into these different Parkinson’s-implicated genes and proteins. There is much reason to hope that within a few years we’ll have the same rich clinical portfolio of drugs against these targets that we do today against alpha-synuclein.
Genetic targets are vital to advancing our mission, but they are far from our only avenue of pursuit; more shots on goal increase our likelihood of success. Additional trials are in progress against other cellular targets, testing compounds that may slow PD progression. MJFF supported early investigations of isradipine (a high blood pressure drug) and inosine (a precursor to the metabolite urate in our cells), and now the National Institutes of Health is funding Phase III studies of both.
While these advances in drug development are cause for cautious optimism, they also highlight a great challenge. Lack of a biological marker of Parkinson’s disease prevents efficient and confident clinical testing of even the most promising therapies.
MJFF has prioritized the identification of PD biomarkers since our earliest days, and 2015 marks the five-year anniversary of our landmark study toward that end. The Parkinson’s Progression Markers Initiative continues to follow volunteers and is enrolling new participants. Initial results are shedding light on potential biomarkers and teaching us more about the nuances of this variable disease.
This year we attained several vital mile markers on the road to a cure, but there is a ways to go. With your support, the Foundation will keep working hard to accelerate our pace, clear the roadblocks and together, reach our destination.
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