Journal
PARKINSONISM & RELATED DISORDERS
Volume 73, Issue -, Pages 105-116Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.parkreldis.2020.04.014
Keywords
Tauopathy; Progressive supranuclear palsy; Treatment; Immunotherapy; Gene therapy
Categories
Funding
- Clinical Research Training Scholarship in Parkinson's disease grant from the American Academy of Neurology/American Brain Foundation/Parkinson's Foundation [2059]
- National Institutes of Health [5P50AG005131-33, 2R01AG038791-06A, U01NS090259, 1U54 NS 092089, U01NS100610, U01NS80818, R25NS098999, P20GM109025]
- Parkinson Study Group
- Michael J Fox Foundation
- Lewy Body Association
- AVID Pharmaceuticals
- Abbvie
- Biogen
- Roche
- NATIONAL INSTITUTE ON AGING [U01NS100610] Funding Source: NIH RePORTER
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Progressive supranuclear palsy (PSP) is a complex clinicopathologic disease with no current cure or disease modulating therapies that can only be definitively confirmed at autopsy. Growing understanding of the phenotypic diversity of PSP has led to expanded clinical criteria and new insights into etiopathogenesis that coupled with improved in vivo biomarkers makes increased access to current clinical trials possible. Current standard-ofcare treatment of PSP is multidisciplinary, supportive and symptomatic, and several trials of potentially disease modulating agents have already been completed with disappointing results. Current ongoing clinical trials target the abnormal aggregation of tau through a variety of mechanisms including immunotherapy and gene therapy offer a more direct method of treatment. Here we review PSP clinicopathologic correlations, in vivo biomarkers including MRI, PET, and CSF biomarkers. We additionally review current pharmacologic and non-pharmacologic methods of treatment, prior and ongoing clinical trials in PSP. Newly expanded clinical criteria and improved specific biomarkers will aid in identifying patients with PSP earlier and more accurately and expand access to these potentially beneficial clinical trials.
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