The world’s largest clinical trial for Parkinson’s disease has commenced, aiming to identify treatments that can slow or halt disease progression
The world’s largest clinical trial targeting Parkinson’s disease progression has officially launched across the UK. The £26 million Edmond J Safra Accelerating Clinical Trials in Parkinson’s Disease (EJS ACT-PD) trial, led by University College London (UCL) and Newcastle University, aims to identify treatments that can slow or stop the disease’s progression. With over 1,600 participants enrolled across more than 40 hospitals, the trial employs an innovative, flexible design to test multiple treatments in parallel, potentially reducing the time needed to evaluate new drug candidates.
Parkinson’s disease and the need for new treatments
Parkinson’s disease is one of the world’s fastest-growing neurological conditions. Around 166,000 people in the UK live with the disease, which progressively worsens over time. Current treatments for symptoms become less effective over prolonged periods, making it vital to find therapies that can slow or halt disease progression.
Innovative trial design
The trial employs a multi-arm, multi-stage design, allowing multiple treatments to be tested simultaneously. This contrasts with traditional trials, where a single group of participants takes a placebo, a method not previously used for Parkinson’s. Initially, the trial will test two drugs: a blood pressure medication and an enlarged prostate drug, both already known to be safe and effective for other conditions.
By analysing results on an ongoing basis, ineffective treatments can be removed, allowing promising drugs to advance more quickly. The trial’s design also permits new treatment arms to be added within the same trial framework.
Expert commentary
Co-chief investigator Professor Thomas Foltynie, from UCL’s Queen Square Institute of Neurology and consultant neurologist at UCLH’s National Hospital for Neurology and Neurosurgery (NHNN), said: “Parkinson’s disease is the second most common neurodegenerative disease worldwide, yet there are no treatments that can slow its relentless progression. We are prioritising drugs that already show promise based on extensive prior evidence, as we seek to identify a treatment that does more than just relieve symptoms. We hope this trial will serve as a blueprint for future research in Parkinson’s and other neurodegenerative conditions.
“Our trial’s wide UK coverage ends the postcode lottery for clinical research, enabling underserved populations with Parkinson’s to participate in this highly inclusive study.”
Professor John Simpson, Director of the MRC-NIHR Efficacy and Mechanism Evaluation (EME) Programme, one of the study’s funders, said: “The next stage of this innovative trial represents a landmark moment in the search for new treatments for Parkinson’s disease, helping improve quality of life for patients.
“This world-leading trial allows experts to test multiple treatments simultaneously to accelerate the discovery of new drugs. Collaboration with other research funders is crucial for enabling innovative and ambitious studies like this, demonstrating how research drives the UK’s life sciences sector and technological leadership.”
Patient perspective
Graham Edwins, the trial’s first participant, said: “I wanted to take part in EJS ACT-PD because of the pioneering approach to test multiple medications in a single trial. Having Parkinson’s, especially young onset, leaves you with choices: denial, acceptance, or to fight back, which is what I feel I am doing by participating. Even if I don’t directly benefit, if I can help progress a potential treatment or cure for the next person diagnosed in their prime, then it’s a job well done.”
