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Linked Clinical Trials – The Development of New Clinical Learning Studies in Parkinson's Disease Using Screening of Multiple Prospective New Treatments

Article type: Review Article

Authors: Brundin, Patrik | Barker, Roger A. | Conn, P. Jeffrey | Dawson, Ted M. | Kieburtz, Karl | Lees, Andrew J. | Schwarzschild, Michael A. | Tanner, Caroline M. | Isaacs, Tom | Duffen, Joy | Matthews, Helen | Wyse, Richard K.H.

Affiliations: Center for Neurodegenerative Science, Van Andel Institute, MI, USA | Cambridge Centre for Brain Repair, Cambridge, UK | Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University Medical Center, Nashville, TN, USA | Johns Hopkins University, Institute for Cell Engineering, Baltimore, MD, USA | University of Rochester Medical Center, Center for Human Exp. Therapeutics, Rochester, NY, USA | Reta Lila Weston Institute of Neurological Studies, University College London, London, UK | Department of Neurology, Massachusetts General Hospital, Boston, MA, USA | The Parkinson's Institute and Clinical Center, Sunnyvale, CA, USA | The Cure Parkinson's Trust, UK. The Pavilion, Mickelfield Hall, Sarratt, Herts, UK

Note: [] Founding Members of the Linked Clinical Trials International Committee. Correspondence to: Patrik Brundin, M.D. Ph.D., Associate Director, Van Andel Research Institute, Director, Center for Neurodegenerative Science, Van Andel Institute, 333 Bostwick Ave. N.E., Grand Rapids, Michigan 49503-2518, USA. Tel.: +1 616 234 5684; Fax: 616 234 5129; E-mail: [email protected]

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Note: [] Founding Members of the Linked Clinical Trials International Committee.

Keywords: Drug repositioning, disease modification, neuroprotection

DOI: 10.3233/JPD-139000

Journal: Journal of Parkinson's Disease, vol. 3, no. 3, pp. 231-239, 2013

Published: 2013
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Abstract

Finding new therapies for Parkinson's disease (PD) is a slow process. We assembled an international committee of experts to examine drugs potentially suitable for repurposing to modify PD progression. This committee evaluated multiple drugs currently used, or being developed, in other therapeutic areas, as well as considering several natural, non-pharmaceutical compounds. The committee prioritized which of these putative treatments were most suited to move immediately into pilot clinical trials. Aspects considered included known modes of action, safety, blood-brain-barrier penetration, preclinical data in animal models of PD and the possibility to monitor target engagement in the brain. Of the 26 potential interventions, 10 were considered worth moving forward into small, parallel ‘learning’ clinical trials in PD patients. These trials could be funded in a multitude of ways through support from industry, research grants and directed philanthropic donations. The committee-based approach to select the candidate compounds might help rapidly identify new potential PD treatment strategies for use in clinical trials.

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