Accelerating Parkinson’s drug development: The International Linked Clinical Trials initiative

Impact of the iLCT

The goal of the iLCT programme is to move more potentially disease-modifying treatments into clinical trials for people with Parkinson’s by choosing drugs with the highest potential for success. Over the past 12 years, the iLCT committee has reviewed 171 unique drugs. This has translated into 21 completed and 20 ongoing clinical trials of iLCT-evaluated drugs, taking place around the world. Furthermore, according to the McFarthing et al. 2023 clinical pipeline paper for Parkinson’s, nearly 30% of all potentially disease-modifying treatments for Parkinson’s in active clinical trial up until 31 January 2023 have been evaluated by Cure Parkinson’s iLCT committee.

Two important currently ongoing clinical trials involving iLCT-prioritised drugs are the Cure Parkinson’s and VAI funded phase 3 clinical trials of the diabetes drug exenatide, led by Professor Tom Foltynie at University College London (UCL), and the phase 3 clinical trial of the respiratory drug ambroxol, led by Professor Anthony Schapira at UCL. Additionally, Cure Parkinson’s supported the UP-Study, a phase 2 clinical trial of the liver drug UDCA, which reported it was safe and well tolerated, and provided evidence to encourage further clinical testing in people with Parkinson’s. Most recently, the Cure Parkinson’s co-funded (with VAI and the French Government) phase 2 trial of lixisenatide – another diabetes medicine – published its results, showing the progression of motor symptoms slowed in those taking lixisenatide.

Progress and continued development of the iLCT

Learnings from the iLCT committee have led to the creation of Cure Parkinson’s iLCT Pipeline Research Acceleration Programme. Some drugs that are reviewed by the committee show strong potential for Parkinson’s but lack important evidence to justify progressing them into clinical trial in people with Parkinson’s. Cure Parkinson’s set up this new grant funded programme to commission the preclinical, lab-based research needed to gain the key evidence required.

Overall, since its creation, more than 4700 people with Parkinson’s have participated in iLCT-related clinical trials globally, and this number is expected to grow as more clinical trials are initiated. People with Parkinson’s have always sat at the centre of the iLCT programme in its goal to progress potential treatments from trials into the clinic. From their participation in the iLCT committee meetings and the conversations on the viability and potential of the drugs being discussed to ensure alignment with patient priorities; to their involvement in clinical trials from planning and execution, to actually taking part in the research. It’s through these continued efforts that it is hoped the impact of the iLCT programme will extend beyond increasing the number of clinical trials to seeing new and varied treatment options being made available in the clinic and, eventually, seeing improvements in quality of life for people with Parkinson’s.

In the meantime, Cure Parkinson’s is also working hard to ensure if a drug proves effective in clinical trials, it will be ready for regulatory approval. One way to do so is by collaborating with regulators early in this process, which can help build a stronger case for making the drug available. We hope these efforts will help to accelerate not only the regulatory process, but also the time it takes to get these therapies to people with Parkinson’s.

It has been my privilege to be involved with the iLCT programme, which has been transformational for Parkinson’s research. The iLCT programme has at its core, principles that underpin its success – patient-centredness, collaboration and transparency. The initiative continues to evolve to meet the needs of the changing international landscape of disease modification trials in Parkinson’s, ensuring its outputs are as relevant and impactful today as they were at its inception.

Professor Camille Carroll, iLCT co-chair

Cure Parkinson’s would like to thank the committee members for volunteering their time and expertise each year, as well as Van Andel Institute and other key funding partners whose support allows the programme to continue.