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The Great Need for Early Diagnostic Tools in the Pursuit of Disease Cures: A Statement from The Michael J. Fox Foundation for Parkinson's Research

In the field of neurodegeneration, we are closing in on the mechanisms of diseases such as Parkinson's and Alzheimer's, but one big thing is keeping cures at arm's length. We need biomarkers to definitively diagnose and track disease, stratify clinical research volunteers and test disease-modifying therapies. And we need tools to identify, validate and screen for biomarkers.

Decisions by pharmaceutical companies and the Centers for Medicare & Medicaid Services (CMS) to cover or reimburse for such tools—neuroimaging scans, for example—have far-reaching implications. In addition to the personal impact for patients, payment for such tests provides incentive to companies to evolve technologies and therapeutics using these tools.

The struggle for coverage of diagnostic testing presents a catch-22: providers will pay if diagnosis and disease tracking can lead to a beneficial treatment, but researchers need data from diagnostic and tracking tools to create such treatments. A decision not to cover scans, tests, biopsies, etc. for diseases still awaiting cures—not just Parkinson's, but the majority of diseases—limits innovation and reinforces the status quo. Millions of patients around the world will tell you, the status quo is not good enough.

Biomarkers: A Bridge to Better Therapies

The role that individual tests play in helping to prevent, halt or reverse disease progression often goes overlooked. The more we can learn about biomarkers such as alpha-synuclein aggregates and beta-amyloid load, the greater knowledge base we have to pull from as we develop drugs to alleviate these problems. Measuring these pathologies earlier in disease will help us create preventive therapies, too. For obvious reasons, biomedical research funding sources require data to support a hypothesis; these tools can provide rationale for investment in new projects.

Furthermore, biomarkers help us test new therapies, speeding the process of getting a new drug to market. Right now, in Parkinson's research, we wait to see if a drug or therapy impacts clinical symptoms; brain scans could show us much faster whether a drug candidate has an effect or not. We also test drugs in a wide patient population, and if the power of effect is not great enough, the medication is sidelined from the trial process. Brain imaging results could help us stratify patients for clinical research to test certain drugs and therapies on those they are more likely to help. This personalized medicine approach has been adopted in cancer with great success. We just don't know how to profile brain disease patients well enough right now, but brain imaging can help us get there.

Some Answers on an Uncertain Disease Course

As they journey through emerging and escalating symptoms, patients rely on a diagnosis and data on disease status to dictate prognosis and treatment plans. We have seen the struggle patients face as they seek a name for what they experience. The empowerment that comes with the ability to tell people what you "have" and what is happening to you should not be underestimated, nor should the stress of uncertainty.

Especially in diseases like Parkinson's and Alzheimer's, where the course of progression varies by individual, tracking the disease can mean more informed decision-making as to treatment options and timing. Alzheimer's now has amyloid and tau tracers that allow researchers to track disease. In Parkinson's we have a scan that can capture dopamine neuron activity, and development of an alpha-synuclein tracer has long been a priority of our Foundation. Today our staff researchers published a peer-reviewed paper in the Journal of Parkinson's Disease on the critical need for a Parkinson's disease tracer and our Foundation's efforts to develop such.

Diagnostic testing on an individual level means better care for that patient today and better treatments for all patients tomorrow. Identification and validation of biomarkers is a critical step toward better quality of life and ultimately a cure for those living with degenerative diseases. We hope that we can call pharmaceutical companies and CMS our partners in fostering an environment that supports the forward-thinking innovation required as we pursue vital biological markers.

Despite the obstacles we face, we are optimistic about the future. The cures are within our reach. We just need the right tools to get there.

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