Emerging drugs for progressive supranuclear palsy

ABSTRACT

Introduction: Progressive supranuclear palsy (PSP) is a common cause of atypical parkinsonism and a rapidly progressive disease that greatly burdens both patients and caregivers. Drugs with disease-modifying potential, targeting mechanisms implicated in the disease’s pathogenesis are currently tested in Phase 1 and 2 trials. If proven efficacious, these compounds might provide substantial benefits not only to patients with PSP but to patients with other tauopathies as well.

Areas covered: Drugs in Phase 1 and 2 trials in PSP, and Phase 2 trials in other tauopathies (Alzheimer’s disease) are reviewed.

Expert opinion: The rationale behind the currently tested compounds as well as the tools available to document a treatment effect offer hope for a therapeutic breakthrough in PSP. The current lack of sufficiently validated biomarkers remains a hurdle that needs to be overcome, in order to facilitate both clinical trials and the accurate prescription of future treatments.

KEYWORDS:

• Clinical trials
• parkinsonism
• Progressive supranuclear palsy
• tau targeted therapeutics
• Tau immunotherapy
• tauopathies
• treatment

Declaration of interest

M Stamelou has received travel and speaker honoraria from Biogen, Specifar and Abbvie, served the advisory board of Biogen, royalties from Oxford and Cambridge University Press, and serves in the editorial board of movement Disorders Journal. M Stamelou is PI in the Passport study funded by Biogen. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.