TUDCA-ALS is a European Consortium (funded by the European Commission under the Horizon 2020 grant) involving top researchers from seven countries across Europe (see Figure 1). The Consortium aims to find a new treatment to slow down the progression of MND/ALS by conducting a clinical trial to investigate the safety and efficacy (effectiveness) of Tauroursodeoxycholic acid (also known as TUDCA).
TUDCA is a molecule that might help to protect motor neurons by camouflaging stress chemicals that trigger a cascade resulting in neurodegeneration (damage and death of the motor neurons).
With this clinical trial, TUDCA-ALS will compare participants who have been randomly assigned to either take TUDCA or placebo (a drug that looks, tastes and smells the same as the drug being tested, but does not contain any active ingredient) over a period of 18 months. All participants enrolled in the study will have a 50% chance of receiving TUDCA or placebo (known as a clinical trial with a ratio of 1:1). The clinical trial will be double-blinded, which means that neither the participants taking part in the study, nor the staff that are running the study will know who will be taking TUDCA or who will be taking the placebo. All participants will also receive the drug riluzole alongside the study treatment.
The main goal is to demonstrate that TUDCA is safe and effective, and to confirm the findings from a previous clinical trial that suggested that TUDCA slows the progression of neurodegeneration in patients with ALS.
Biological samples (blood and cerebral spinal fluid) will also be taken during the TUDCA-ALS Clinical Trial, to enable their secondary goal – looking at a number of biomarkers (characteristics of biological change over time) that are associated with disease progression.
TUDCA-ALS will also look at other biological variants to determine whether there are some differences that allow patients to respond better (or indeed worse) to the treatment. This could be used to move away from a ‘one size fits all’ type of therapy to ‘personalised medicine’.
The Consortium are currently working on the arrangements and approvals that may allow study participants the opportunity to continue onto an open label phase of the study at the end of the clinical trial, where they will be given TUDCA regardless of whether they were in the TUDCA or placebo arm of the clinical trial.
The TUDCA-ALS Consortium started its project in January 2018, and have been working hard performing all of the preparatory activities, obtaining all of the necessary approvals and training all the involved staff, so that recruitment into the TUDCA-ALS Clinical Trial could begin.
The first participant was recruited into the TUDCA-ALS clinical trial by the lead partner, Humanitas, at the start of the year and we are very pleased to announce that the UK is now ready to start recruiting participants into the TUDCA-ALS trial. There are seven sites in the UK and Ireland that will be recruiting participants into the TUDCA-ALS clinical trial: Dublin, Liverpool, Preston, Plymouth, Salford, Sheffield and Stoke (see Figure 2).
This project has received funding from the European Commission’s Horizon 2020 research and innovation rare diseases and orphan drug programme under grant agreement No 755094. This output reflects the views only of the author(s), and the European Union cannot be held responsible for any use which may be made of the information contained therein.