Parkinson’s Disease at 200 – Scientific American Blog Network – Scientific American (blog)

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.

Its 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.

Its 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, hed 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, hed 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 Parkinsons, nor to conclusively track its progression over time. Make no mistakeweve made tremendous progress toward our goal, but we are realistic about the work that remains ahead of us.

As we mark Parkinsons Awareness month, 200 years after James Parkinson started the clock on scientific inquiry into PD, I want every Parkinsons 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 doesnt, 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 (wheres 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 playersindustry, government, researchers and non-profit groupsare 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 Parkinsons 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:

This state of affairs represents a dramatic shift from only about 15 years ago when Michael J. Fox launched the foundation. The Parkinsons drug pipeline was stalled; stem cells hadnt 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 Parkinsons research, and the participation of enterprising nonprofits in the drug development ecosystem was still a curious novelty.

Today, the patient communitys active involvement in our mission holds unprecedented potential to build Parkinsons 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 were 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 Parkinsons; 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 isnt necessarily cause for celebration; wed rather achieve our mission and go out of businessfast. Our goal is to cure Parkinsons, and with the participation of a robust and empowered community, weve never been closer. Thats because, as Michael J. Fox would say, The only thing thats incurable is our optimism.

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Parkinson's Disease at 200 - Scientific American Blog Network - Scientific American (blog)