UK starts 'largest-ever' trial of possible Parkinson's drugs

Researchers in the UK have started recruiting patients for a large-scale trial that will test a battery of Parkinson's therapies to see if they can slow down or halt the progression of the disease.

The basket trial approach – which came to the fore during the COVID-19 pandemic a few years ago – will be applied to Parkinson's in the new £26 million ($35 million) study – called EJS ACT-PD – which aims to enrol around 1,600 patients.

The approved protocol for EJS ACT-PD will initially test two well-established drugs – blood pressure therapy telmisartan and terazosin for enlarged prostate – to see if they can be repurposed as Parkinson's treatments, comparing them to placebo.

A third drug – liver disease treatment ursodeoxycholic acid (UDCA) – will start testing next year, and other candidates may also be added, according to the lead investigators, based at University College London and the University of Newcastle.

By testing more drugs more efficiently than ever before, the trial could take up to three years off the time needed to test a drug candidate, they said.

In a flexible trial design, the protocol will allow patients who fail to show a benefit with one drug to switch to another, with follow-up – in-person or remotely – every six months for up to three years. It will also aim to recruit a population representative of the Parkinson's population in the UK, covering people with diverse backgrounds, varying ages above 30, and a range of treatment histories.

Moreover, the aim is that the infrastructure and network of sites delivering the study will become largely permanent, unlike conventional studies in which it is dismantled after completion, so testing of additional therapies can be rolled out quickly.

"We are prioritising drugs that already show promise as potential treatments, based on an extensive review of prior evidence, as we seek to identify a drug that does more than just provide symptom relief for Parkinson's," said Prof Thomas Foltynie of UCL, who is co-chief investigator for the study alongside Newcastle's Prof Camille Carroll.

"We hope this trial will serve as a blueprint for future trials in Parkinson's and other neurodegenerative conditions," he added.

Trial participants are already being recruited in London and Newcastle, and between now and April next year, trial sites at more than 40 hospitals across the four UK nations will come online, according to the organisers

The first patient to be recruited, Graham Edwins, said: "Having Parkinson's, especially young onset, your choices are denial, acceptance, or to fight back, which is what I feel I am doing by taking part. 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."

Parkinson's disease is one of the world's fastest-growing neurological conditions, with 166,000 people affected in the UK alone.

The study is being sponsored by UCL and funded by an MRC and NIHR partnership, Cure Parkinson's, The Michael J Fox Foundation, Parkinson's UK, The John Black Charitable Foundation, The Gatsby Charitable Foundation, and Van Andel Institute.

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