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Seeking progress in disease modification in Parkinson disease
Codrin Lungu1, Jesse M Cedarbaum2, Ted M Dawson3
1Division of Clinical Research, National Institute of Neurological Disorders and Stroke, National Institutes of Health, 6001 Executive Blvd, #2188, Rockville, MD, 20852, USA.
Parkinsonism & Related Disorders
|September 25, 2021
Summary
Achieving disease modification in Parkinson disease (PD) is challenging. This review identifies barriers and proposes research priorities, including biomarkers and novel trial designs, to improve success rates for PD treatments.
Area of Science:
- Neuroscience
- Neurology
- Clinical Trials
Background:
- Disease modification in Parkinson disease (PD) remains an unmet clinical need despite extensive research.
- Past therapeutic development efforts have not yielded disease-modifying treatments for PD.
Purpose of the Study:
- To review the current state of scientific understanding regarding disease modification in Parkinson disease.
- To analyze reasons for the failure of previous clinical trials to demonstrate disease-modifying effects.
- To propose solutions and research priorities for future PD therapeutic development.
Main Methods:
- Convened a meeting with leaders in the field and key stakeholders, including representatives from the National Institute of Neurological Disorders and Stroke (NINDS).
- Discussed drug therapy strategies aimed at achieving disease modification in Parkinson disease.
Main Results:
- Identified critical areas for future research: development of relevant biomarkers, refinement of patient population selection, novel clinical trial designs, and enhanced stakeholder collaboration.
- Highlighted differences in therapeutic target and agent selection processes across various development organizations.
Conclusions:
- Progress in Parkinson disease modification is hindered by several barriers.
- Proposed research priorities are essential to increase the likelihood of successfully developing disease-modifying therapies for Parkinson disease.
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