“Major step” in Parkinson’s research as new staging framework announced

Advances Global update Research

Author: Laura Vickers-GreenPublished: 25 January 2024

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Leading scientists and patient organisations have worked together to release a research framework for defining and staging Parkinson’s based on biology, rather than clinical symptoms. The impact of this framework could accelerate research, improve the development of new drugs and help diagnose Parkinson’s before physical symptoms emerge.


An international working group of Parkinson’s experts and patient organisations has proposed a significant new research framework that – for the first time – stages Parkinson’s and defines it based on its underlying biology.

This new framework has been published in a paper in the January issue of The Lancet, after being developed by the Critical Path for Parkinson’s Consortium (CPP), which was founded by the Critical Path Institute and Parkinson’s UK, with collaboration from organisations including The Michael J Fox Foundation and Parkinson’s Europe.

What is the new Parkinson’s framework?

The new proposal outlines how two medical tests could be used to accelerate research by more accurately identifying Parkinson’s in clinical trials. The two tests are:

  • a new test that can identify a protein called alpha-synuclein (which Parkinson’s causes to clump together, damaging the brain) in spinal fluid taken via a lumbar puncture – making this test the earliest known indicator of Parkinson’s
  • a brain scan called a DaTSCAN that can tell if there is a lack of a chemical called dopamine in the brain, another crucial sign of Parkinson’s

Importantly, both tests can help to identify Parkinson’s in people even before symptoms emerge, which The Michael J Fox Foundation describes as a ‘paradigm shift after nearly two centuries of relying on outward, primarily movement-based symptoms’ to detect the condition.

This new biological framework for Parkinson’s can then be used in a system for staging the disease that accounts for Parkinson’s risk, diagnosis, and functional impairment ranging from slight to severe. A person’s Parkinson’s “stage” is based on a combination of their genetic risk factors and the results of the two tests above (i.e the presence of alpha-synuclein in the spinal fluid, and depleted levels of dopamine in the brain).

Accelerating research

While this new staging system isn’t being used for Parkinson’s treatment just yet, the framework will help with clinical trials. Claire Bale from Parkinson’s UK explains how:

‘A huge challenge for Parkinson’s clinical trials is that the condition is currently diagnosed and monitored based on symptoms which can vary from person to person, and from hour to hour.

For trials to be successful, it is important that the right people are identified to take part, and that we can measure whether the treatment has the desired effects.

Having tests that can tell us what is happening inside the brain has the potential to revolutionise clinical trials. They will allow us to select the right people and better measure the potential of the treatment under investigation.’

Being able to begin clinical trials in people with Parkinson’s who aren’t even showing physical symptoms yet could ultimately lead to therapies that prevent the onset of those symptoms entirely.

Just the beginning

This new framework is an exciting milestone for Parkinson’s, but members of the Critical Path for Parkinson’s Consortium view it as the starting point in an ongoing effort to develop the scientific knowledge that will help to identify, treat and ultimately cure Parkinson’s.

Peter DiBiaso, co-author of the paper and member of The Michael J Fox Foundation’s Patient Council, says:

‘It’s still early, but this framework will have an immediate impact in terms of how we’re designing clinical protocols and optimising research that can lead to better treatments that patients are waiting for. We know there’s a lot of work to be done, but this is the most important first step the field can take together to rapidly advance breakthroughs for patients and families.’

Professor David Dexter, Director of Research at Parkinson’s UK, says

‘This initial framework is just the beginning. These two tests are a great start but there are lots of exciting advances happening in this field at the moment… The ultimate goal is to accelerate the development of new treatments that can transform the lives of people with the condition.’

And finally, Tanya Simuni, MD< – lead author of the research paper and director of the Parkinson’s Disease Movement Disorders Center at Northwestern University – says:

“Our shared hope is that this new framework will foster innovation in clinical development, making trials more efficient and streamlining regulatory review… The success that the Alzheimer’s field has had with its biological framework provides the inspiration and motivation to achieve similar accelerated timelines in Parkinson’s. Ten years from now, we hope we will look back and say this framework was the key that finally opened the door to next-generation treatments in Parkinson’s.”

Read the full research paper on The Lancet

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