Linked Clinical Trials

The research record

This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".

Who

Patrik Brundin, Roger Barker, P. Jeffrey Conn, Ted Dawson, Karl Kieburtz, Andrew Lees, Michael Schwarzschild, Caroline Tanner, Tom Isaacs, J. Duffen, H. Matthews and Richard Wyse. Isaacs and Wyse were at the Cure Parkinson's Trust (CPT), the charity that conceived and organised the programme. Initial planning began in 2010; the committee was set up in 2011 and first met in 2012 at the Van Andel Institute, Michigan (Wyse et al. 2024).

What happened

Developing a new drug takes many years. Repurposing a drug already in use can skip early safety work. The group first identified 72 candidate approaches, narrowed to 26 for reasons of safety, brain entry or patents, and wrote a dossier on each (biology, animal data, human data). The 26 included GLP-1 agonists, a DPP-4 inhibitor (sitagliptin), metformin, iron chelators, a statin, trehalose, rapamycin and nilotinib. An expert committee scored them and prioritised some for pilot trials: exenatide, liraglutide, lixisenatide, deferiprone/deferasirox, simvastatin and trehalose were named. The abstract says 10 were worth moving forward; the body text says 7 (6 drugs and one natural compound) for immediate pilots, with 5 on a waiting list. The paper does not reconcile the two. Journal of Parkinson's Disease 3(3):231-239, 2013.

The committee has met yearly. By 2024 it had evaluated 171 unique agents, with 21 completed and 20 ongoing trials linked to the initiative (Wyse et al. 2024). A 2021 review of the programme (Stott, Wyse, Brundin) describes seven completed and 15 ongoing trials of 16 agents. The 2021 review lists the outputs: exenatide, ambroxol, ursodeoxycholic acid, simvastatin (PD-STAT: safe, no disease change), deferiprone and nilotinib.

• Funder report (source sweep, Oct 2026). Cure Parkinson's reports that its 13th iLCT meeting (Grand Rapids, in the year to March 2025) evaluated 17 dossiers and prioritised 5 compounds for clinical testing. It counts 17 iLCT-evaluated drugs in 19 active trials (3,766 people) and 20 drugs in 28 completed trials (3,108 people), and says the programme has helped draw more than £100 million in outside funding. The 31% share of disease-modifying trials it cites comes from the 2024 McFarthing pipeline review, not read here.

Why it moved us toward a cure

It gave a transparent way to choose which compounds deserve scarce patient time, and tied lab evidence to clinical trials. It produced the lines that became the GLP-1/exenatide story (landmark 20), ambroxol and deferiprone, and its logic led to the shared-placebo platform EJS ACT-PD.

Outcome

Advanced as a process, with a mixed hit rate: exenatide Phase 3, deferiprone and nilotinib (landmark 68) did not slow disease; ambroxol is in Phase 3. Caveats: deliberations are closed, the published reviews are written by committee members, and many pilot trials were small or open-label.

Sources

• Brundin et al., J Parkinsons Dis 2013;3:231-239, DOI 10.3233/JPD-139000
• Stott, Wyse, Brundin, Front Neurosci 2021;15:653377
• Wyse et al., "Twelve years of drug prioritization," J Parkinsons Dis 2024;14:657-666, DOI 10.3233/JPD-230363
• Cure Parkinson's iLCT page
• Cure Parkinson's Impact Report 2025 (1 Apr 2024 to 31 Mar 2025)
• Not yet verified: the affiliations and roles of Duffen and Matthews; which 2021 outcomes later changed (the review predates the exenatide Phase 3 result). No correction or retraction was found in Crossref or Europe PMC.