Two centuries after its discovery, it's still incurable—but research is accelerating, with major help from citizen scientists in the patient community
Michael J. Fox, diagnosed in 1991, has been a tireless crusader for Parkinson's research Credit: Paul Hudson Flickr (CC BY-SA 2.0)
2017 marks 200 years since James Parkinson, an English apothecary, surgeon, geologist and sometime political activist, published a paper that defined his legacy. An Essay on the Shaking Palsy described a syndrome that caused wide-ranging problems that affected how people move, feel, speak, think and even look. This constellation of symptoms was probably as old as the human race, but Parkinson was the first to scientifically characterize it. Today we know it as his disease.
It’s estimated that five million people worldwide are currently living with Parkinson's disease, or PD, which we now know kills the neurons that produce dopamine (a critical chemical in brain function) among other changes in the brain. Most people get it around age 60, though it is also seen in people as young as their 20s. The disease is progressive, meaning that it gets inexorably worse over time, and many live with it for decades. Estimated prevalence numbers are likely on the low side because PD is frequently misdiagnosed and can lead to isolation and social withdrawal.
It’s interesting to imagine what Dr. Parkinson would think about what two centuries of scientific exploration have done to bring us closer to a cure for PD.
On one hand, he’d be amazed by the research breakthrough that led to the 1967 discovery of levodopa, which 50 years later remains the “gold standard” Parkinson's treatment. On the other, he’d likely be surprised to learn that doctors continue to diagnose PD much in the same way he did in his day: there is no objective test or biomarker to predict, prevent or diagnose Parkinson’s, nor to conclusively track its progression over time. Make no mistake—we’ve made tremendous progress toward our goal, but we are realistic about the work that remains ahead of us.
As we mark Parkinson’s Awareness month, 200 years after James Parkinson started the clock on scientific inquiry into PD, I want every Parkinson’s family to know they play an extremely vital role in speeding development of promising treatments. They are the real experts on the disease, with strong points of view on what works and what doesn’t, as well as what needs are still unmet.
A number of converging factors have created an unprecedented new era of patient engagement that promises to launch all of our past efforts into hyper-drive (where’s that flux capacitor when you need one?). Inspired to action by vocal advocates such as Muhammad Ali and Michael J. Fox, who started the research foundation I currently helm, this community has joined forces so that all key players—industry, government, researchers and non-profit groups—are now at the table alongside patients who are actively engaged in their own health and helping to find a cure.
As the leader of a foundation dedicated to accelerating research breakthroughs and a cure for Parkinson’s disease, I can say with confidence that the PD field has made greater progress over the past two decades than in the two centuries that came before. To name just a few heartening examples:
- The field of genetics research has opened doors to a new understanding of the disease and greatly increased opportunities to slow, stop or even reverse its progression.
- What patients always knew but researchers have recently come to understand is that the well-known movement symptoms of PD are merely the tip of the iceberg, and ongoing studies are now addressing undertreated non-motor symptoms, including pain, fatigue and cognitive and mood changes.
- Risk factors including loss of the sense of smell, certain sleep disorders and even ethnicity are helping us identify and study at-risk populations to devise strategies for earlier diagnosis and treatment.
- New hypotheses have yielded evidence that the side effects of treatment may be lessened by changing how we deliver it—leading to the development of skin patches, inhalers and other devices that could vastly improve patients’ quality of life.
- Longitudinal observational studies are bringing us closer than ever to the development of biomarkers, thanks to the dedication of researchers worldwide and the participation of thousands of people with PD.
- A handful of new therapies have come to market in the past couple of years. Several more are in late-stage testing, with a few expected to gain FDA approval well before Parkinson’s hits its 205th anniversary.
This state of affairs represents a dramatic shift from only about 15 years ago when Michael J. Fox launched the foundation. The Parkinson’s drug pipeline was stalled; stem cells hadn’t panned out as the silver bullet many had hoped for, and little else was in R&D pipeline. Few incentives existed for pharmaceutical companies to invest in Parkinson’s research, and the participation of enterprising nonprofits in the drug development ecosystem was still a curious novelty.
Today, the patient community’s active involvement in our mission holds unprecedented potential to build Parkinson’s research momentum to warp speed. Emerging technologies have created vast opportunities for “citizen scientists” to contribute data, report on health and treatment outcomes, correct long-held myths about PD, and better inform efforts by scientists, regulators and payers to shape the results patients want and need.
The research highway keeps adding more lanes, and we’re determined to help everyone navigate to the right on-ramp. That means different things to different people: participating in traditional clinical trials or taking part in virtual and online research opportunities, for example. Some will advocate for policies that help everyone touched by Parkinson’s; others will simply want to get educated and stay up to date on the evolving science of this complex disease.
Two hundred years is a long time, and this milestone isn’t necessarily cause for celebration; we’d rather achieve our mission and “go out of business”—fast. Our goal is to cure Parkinson’s, and with the participation of a robust and empowered community, we’ve never been closer. That’s because, as Michael J. Fox would say, “The only thing that’s incurable is our optimism.”
https://blogs.scientificamerican.com/guest-blog/parkinsons-disease-at-200/
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