Precision ALS, which is led by two Irish research centres – ADAPT and FutureNeuro – involves world class Irish-based researchers in clinical science, data science and artificial intelligence (AI).

The researchers will work in partnership with TRICALS, an independent consortium of leading ALS experts, patients and patient advocacy groups across Europe.  National and international industry partners and charities including patient organisations are also actively participating.

The research is supported by the Irish government through a Science Foundation Ireland (SFI) investment of €5m, which will be leveraged with an additional €5m from industry partners.

Leo Varadkar, Tánaiste and Minister for Enterprise, Trade and Employment, said: “This project straddles clinical research and industry, and will combine the best of our technologies, the best of our ideas, and the best of our medical expertise with the potential to change lives for the better.

'Supporting job creation and reducing drug costs'

"It will develop tools that facilitate clinical trials based on precision medicine and has the potential to produce benefits for other rare conditions and diseases, supporting job creation and reducing drug costs.”

Precision ALS will provide an innovative and interactive platform for all clinical research in ALS across Europe, that will then harness AI to analyse large amounts of data.

As the largest international multimodal dataset aimed at precision medicine for this condition, Precision ALS will address the issues with gathering new data at scale in a timely and cost-effective manner across multiple international sites to present that data in real time to clinical scientists.

Dr Linda Doyle, provost of Trinity College Dublin, said: “The Precision ALS programme builds on a dynamic culture of research innovation here in Trinity. It highlights the synergies that exist between advanced technology and clinical research and the potential they have to advance not just our understanding of disease but also the treatment of it.  

"This collaboration will train a cohort of highly skilled, industry ready researchers, and generate new jobs in clinical and data science research. It will strengthen Ireland’s position as a global leader in MND research and AI. I wish all involved the very best.”

'Different patterns of progression'

Director of the Precision ALS research programme and professor of neurology at Trinity, Professor Orla Hardiman, based in the Trinity Biomedical Sciences Institute, said: “Despite significant advances in pre-clinical models that help us understand the biology of disease in animals, the success of clinical trials has been disappointing.

"ALS is a disease that only affects humans, and there is increasing recognition of the need for a precision medicine approach towards drug development. We know now that ALS is heterogeneous, meaning that it has different causes and different patterns of progression.

"Large numbers are required to understand these differences. Using ‘big data’ analyses, Precision ALS will provide an in-depth understanding of the factors that drive heterogeneity, and in doing so will for the first time allow us to target new and innovative treatments to specific patient subgroups.”

ALS/MND causes progressive decline in movement, cognition and behaviour. Although uniformly terminal, life expectancy can vary from three months to many years from first symptom, and there are no effective treatments.

Irish researchers, along with their European collaborators in ALS/MND, have shown that the disease is caused by variable combinations of faulty genes that likely interact with lifestyle and environment.

Using big data analysis, Precision ALS will provide the technology to improve our understanding of how these factors impact the development of the disease. This in turn will inform which treatments will work for each individual, instead of a one-size-fits-all approach.

From a computational and data-science perspective, Professor Vinny Wade, director of the SFI ADAPT Centre for AI-Driven Digital Content Technology at Trinity, said: “Precision ALS brings together a perfect mix of data and technology research skills to trailblaze discoveries in tackling these devastating diseases.

"Our experience in researching these datasets for immediate interrogation using AI will help identify contributing factors and increase our power to discover changes linked to disease.  

"Unlocking this data in an ethical way is the key to achieving the research mission and realising true ‘precision medicine’.  This pioneering work will lead to transformational change for patients with a ripple effect that will positively impact society.”

Professor of neurology and chair of TRICALS, Leonard van den Berg, said: “For the last 10 years TRICALS partners have worked closely together to generate a large amount of data relating to ALS. We are delighted to be the strategic partner with ADAPT and FutureNeuro.

"This programme has the ambition to break new boundaries to develop targeted therapies, and to provide the right drug for the right patient at the right time. This has the potential to have a hugely positive impact on the lives of those with ALS.”

Professor Philip Nolan, director general of SFI, said: “The SFI Research Centres were developed to create critical mass of excellent research in areas of national importance, and to ensure this research has tangible benefits for our health, our society and our economy through collaboration between academia, government and industry across the island of Ireland and internationally.”

Simon Harris, Minister for Further and Higher Education, Research, Innovation and Science, said: “The Covid-19 pandemic has taught us the value of research and the difference it can make to people’s lives. This is a perfect example of the impact research and innovation can have. By supporting and harnessing these types of advanced research projects we will ultimately see the benefits across society.” 

Better clinical outcomes

Precision ALS is a unique programme that brings together clinicians, computer scientists, information engineers, technologists, and data scientists.

The researchers will work together with a number of companies including Biogen, Novartis, Takeda, IQVIA, Roche and Accenture to generate a sustainable precision medicine-based approach towards new drug development that will have many benefits including better clinical outcomes for patients and reducing the economic cost of these diseases.

Professor David Henshall, director of the SFI FutureNeuro Centre for chronic and rare neurological disease and Professor of Molecular Physiology and Neuroscience at RCSI University of Medicine & Health Sciences, said: “The launch of Precision ALS marks an important step towards achieving FutureNeuro’s mission to change the patient journey for high-burden neurological disease.

"By working with ADAPT, this programme integrates frontier basic science, applied clinical research and cutting-edge data science to enable a precision-medicine approach towards new drug development for ALS.”

On completion, Precision ALS will be a first-in-kind modular transferable pan-European ICT framework for ALS that can be easily adapted to other diseases that face similar precision medicine-related challenges.