An update to our research strategy
Every few years, we set out to review and refresh our research strategy. This document underpins everything we do at Cure Parkinson’s and provides the roadmap for how we plan to move closer to our goal: to identify curative treatments for Parkinson’s, with urgency.
As part of this update, we have identified four key objectives for the next five years. Learn more about each of these objectives and the efforts we are making to accelerate the discovery of a cure below.
1. Grow our treatment selection programme
Identifying promising treatments is a difficult but essential task. Clinical trials require a substantial amount of time and resources to run, therefore, it is important that we are investing in drugs with the highest probability of success, and designing studies that will produce the results needed to decide whether to continue its investigation. To aid in this process, Cure Parkinson’s and Van Andel Institute launched the International Linked Clinical Trials (iLCT) initiative in 2012. Each year, a committee of 20-30 Parkinson’s experts from around the world meet to prioritise promising drugs for clinical trial.
For the past 13 years, the iLCT committee has reviewed over 200 potentially disease-modifying treatments for Parkinson’s. Now, we seek to further expand this initiative. One way we are doing so is by encouraging cross-condition collaborations. Many neurodegenerative conditions, including Parkinson’s, Alzheimer’s, and Multiple Sclerosis, have common underlying biological features. This means that treatments for one may also have benefits in another. At this year’s iLCT meeting, Cure Parkinson’s partnered with Alzheimer’s Research UK to hold a special joint session, which involved a separate committee of Parkinson’s and dementia experts reviewing several drugs with the potential to treat both conditions. Over the next five years, we endeavour to set-up similar collaborations and encourage knowledge sharing between conditions.
2. Accelerate the clinical testing of new therapies
Setting up clinical trials is an expensive, time-consuming process. It is like building a football stadium for a single match, tearing it down, then rebuilding it for the next one. To accelerate this process, Cure Parkinson’s has aided in the funding and development of several multi-arm clinical trial platforms – trials that allow the testing of multiple treatments simultaneously.
One such platform is The Edmond J Safra Accelerating Clinical Trials in Parkinson’s Disease (EJS ACT-PD) trial, which launched earlier this year in the UK. EJS ACT-PD aims to involve 1600 people with Parkinson’s and will be testing two treatments – telmisartan and terazosin – in the first instance. A third treatment group with ursodeoxycholic acid (UDCA) is planned for 2026. Furthermore, EJS ACT-PD is both multi-arm and multi-stage (MAMS), meaning it can facilitate the transition between phases of clinical testing. Cure Parkinson’s is proud to support initiatives like this to aid in our goal of finding curative treatments with urgency.
3. Champion the development of combination therapies
To date, most experimental treatments involve testing a single drug targeting a single aspect of a condition. We know, however, that Parkinson’s has many underlying drivers. Therefore, it may be more effective to treat Parkinson’s with combinations of agents that target different aspects of the condition.
A combination therapy is a treatment that involves two or more active agents to achieve the desired effect. They have been successfully employed in other disease areas, such as cancer, and are even used to treat Parkinson’s symptoms. Co-careldopa, one of the most common Parkinson’s medications, contains both levodopa and carbidopa. Given this, it feels logical that combination therapies could also be used to slow, stop or reverse Parkinson’s progression. To encourage researchers to think about these therapies, Cure Parkinson’s launched a £2m funding call in October to support preclinical and clinical studies of combination therapies.
4. Make disease modification more personalised
Parkinson’s can vary widely between people affected by the condition. This can make it hard to determine the effectiveness of a treatment in a clinical trial, as each person may respond differently to the therapy. To increase the likelihood of success, we are interested in improving patient stratification for clinical trials and helping researchers identify who could benefit the most from a therapy.
One way to do so is by identifying individuals with specific genetic risk factors for Parkinson’s. For example, ambroxol – a cough medicine – increases activity of an enzyme called glucocerebrosidase, or GCase. Variations in the gene GBA1, which produces GCase, are the most common genetic risk factor for Parkinson’s. In the ongoing phase 3 trial of ambroxol (ASPro-PD), half of the participants with have a GBA1 mutation to evaluate whether ambroxol has a greater effect in that population.
Our clear focus means we are relentless in our pursuit of these objectives and the ultimate goal of disease modification for Parkinson’s. We will bring together those who share this ambition and work collaboratively with them as we all strive for a world without Parkinson’s.
Dr Simon Stott, Director of Research, Cure Parkinson’s
