Comment on the results of the CENTAUR trial

The recent announcement of the positive results from the phase 2 CENTAUR trial are exciting and promising for the TUDCA-ALS trial. 

The recent publication of the results from the CENTAUR Trial is of great interest to the TUDCA-ALS consortium. The CENTAUR trial was a phase 2/3 clinical trial looking at a drug named AMX0035, which is a combination of phenylbutyrate and tauroursodeoxycholic acid (TUDCA).

The CENTAUR trial followed 137 participants (89 taking AMX0035 and 48 taking a placebo) in the United States, for a period of 24 weeks. The results suggest that the treatment with AMX0035 has a clinically meaningful benefit and a favourable safety profile for people living with ALS.

The results of the CENTAUR trial are in line with those of a pilot phase 2 study, looking at the safety and efficacy of TUDCA on 60 people with ALS. This study suggests that TUDCA plays a role in delaying the progression of the disease, thus showing great promise for its use as an add-on treatment for ALS’  .

The currently ongoing TUDCA-ALS study is a phase 3 clinical trial supported by the European Commission, which aims to ascertain the long-term effectiveness and safety of TUDCA in people living with ALS. TUDCA-ALS will follow 440 participants in seven countries in Europe. Participants will be taking 1g of TUDCA or matched placebo twice a day, for a total 78 weeks (18 months).

The TUDCA-ALS consortium comments that the recent results from the CENTAUR trial confirm the great potential of TUDCA as an add-on treatment in people living with ALS. Given this recently evolved background, the TUDCA-ALS trial is now even more in the position to provide fundamental answers to ALS patients in Europe and worldwide.

If you would like to learn more about the study and how to take part, please visit

TUDCA-ALS study continues despite COVID-19 outbreak

We would like to reassure you that despite the recent outbreak of COVID-19 epidemics across Europe, that all TUDCA-ALS trial activities are currently ongoing.

There continues to be regular shipments of the tablets that the participants are taking as part of the study (known as the Investigational Medicinal Product or IMP) as well as the shipments of the equipment and materials required to collect biological samples.

Despite the restrictions on travel within Italy, patients are permitted to travel in order to attend medical assessments.
We will of course provide further updates if the situation changes in the future.

TUDCA-ALS recruits first participant

TUDCA-ALS, a phase III clinical trial to determine whether TUDCA (Tauroursodeoxycholic Acid) in combination with riluzole can slow progression of ALS has recruited its first study participant.

The trial plans to recruit 440 participants across 7 countries; Italy, Germany, UK, France, Belgium, the Netherlands and Ireland and will investigate a new drug treatment for amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND), has recruited its first participant in Italy.

The study is organised by the TUDCA-ALS Consortium formed by ten EU partner organisations, led by the research group of Prof. Alberto Albanese, Head of Neurology at Humanitas Mirasole Spa, Italy and is funded by a € 5.6 million funding grant from the European Commission Horizon 2020 Programme.

Professor Albanese says “I am excited to announce that the TUDCA-ALS clinical trial has recruited its first participant in Italy and am looking forward to the trial rolling out across the European network of participating centres in the coming months.”

The aim of the trial is to determine whether the drug TUDCA (Tauroursodeoxycholic Acid) in combination with riluzole can slow disease progression.

TUDCA is a molecule that helps to protect motor neurons by camouflaging a stress chemical released at the start of a process that results in cell death. It is already used to treat diabetes and certain liver conditions by increasing the expression of insulin-degrading enzyme. Its function as treatment that can protect the motor neurons is however still in the developmental stages.

An initial pilot study on 60 people with ALS showed that TUDCA significantly delayed the degeneration of motor neurons by about one third, allowing for longer survival of people living with the disease.

TUDCA-ALS Kick-off meeting in Milan

The ‘TUDCA-ALS Kick-off meeting’ was held on Thursday 8 and Friday 9 February at the Lead Coordinating Site, Humanitas University, and attended by the top international experts on this neurodegenerative disease, for which there is currently no cure.

The meeting marked the official start of this Humanitas-coordinated international clinical study that ranked first amongst the 127 applications to the Horizon 2020 call for rare diseases and orphan drugs, and was awarded €5.6 million by the European Commission. The meeting brought together leading ALS medical specialists from Italy, Ireland, Great Britain, Germany, France, the Netherlands and Belgium.

ALS, also known as Motor Neuron Disease (MND) and Lou Gehrig’s disease (from the name of the famous baseball player whose illness raised public attention in the 1930s), is a progressive neurodegenerative disease of adulthood, caused by the loss of spinal, bulbar, and cortical motor neurons, leading to paralysis of voluntary muscles and eventually death through respiratory insufficiency.

Prof. Alberto Albanese, Head of Neurology at Humanitas said “At the moment, there is no cure for ALS and the few limited treatments currently available slow down disease progression by only a few months. An initial first pilot study on 60 patients recently showed that TUDCA significantly delayed the degeneration of motor neurons in ALS patients by about one third, thus allowing for longer survival of patients”.

TUDCA-ALS website goes live

The TUDCA-ALS website includes information about the project, the partners involved and, as time progresses, will provide information on who to contact if you are interested in joining the TUDCA-ALS Clinical Trial, and any presentations or publications that are produced as a result of the work done by the TUDCA-ALS project.

TUDCA-ALS project begins

The TUDCA ALS consortium, funded through a € 5.6 million funding grant from the European Commission Horizon 2020 Programme, started on 1st January 2018.

TUDCA-ALS has assembled and integrated a network of Europe’s 10 most talented and esteemed academic re-searchers from partner institutions in seven EU countries including the Italian Humanitas Mirasole SpA, the Istituto Superiore di Sanità and SME pharma company Bruschettini of Genoa, and several European academic centres: the University of Ulm (Germany), the University of Sheffield (United Kingdom), the University Hospital of Tours (France), the Catholic University of Leuven (Belgium), the University Medical Center of Utrecht (Netherlands), and Trinity College Dublin (Ireland). The Motor Neurone Disease Association (United Kingdom), is a partnering patient association confirming the primary role played by lay associations in this disease.

The “TUDCA-ALS project” includes the trial of a new drug treatment against Amyotrophic Lateral Sclerosis (ALS).

“The study, led by Humanitas, will last for four years and will allow us to evaluate the therapeutic efficacy of a new treatment for ALS, using a drug derived from bile acids, called Tauroursodeoxycholic Acid (TUDCA)” – said Prof. Alberto Albanese, Head of Neurology at of Humanitas.