€10M European AI Project Hopes to Develop Precision Medicine for ALS

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by Marta Figueiredo, PhD |

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An ambitious Irish research project, called Precision ALS, will combine clinical research, data science, and artificial intelligence (AI) to identify the multiple — and potentially targetable — factors involved in the development and progression of amyotrophic lateral sclerosis (ALS).

The goal is to use this information to determine which treatments will work for each patient, instead of a one-size-fits-all approach.

“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,” Orla Hardiman, MD, the project’s director and a professor of neurology at Trinity College Dublin, in Ireland, said in a press release.

“We know now that ALS is heterogeneous, meaning that it has different causes and different patterns of progression,” and large amounts of data “are required to understand these differences,” she added.

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“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,” Hardiman said.

The project — supported by an investment of €5 million (nearly $5.5 million) from Science Foundation Ireland (SFI) and an additional €5 million from industry partners — will be led by two SFI research centers: ADAPT and FutureNeuro.

The ADAPT center for AI-driven digital content technology is led by Trinity College Dublin, while the FutureNeuro research center for chronic and neurological diseases is hosted by the RCSI University of Medicine and Health Sciences, also in Dublin.

“I am delighted to welcome the launch of Precision ALS,” said Philip Nolan, MD, PhD, SFI’s director general. “This collaboration will directly benefit healthcare and patient communities, and yield new knowledge, approaches and treatments with the potential to improve the lives of many.”

Within the RCSI, Precision ALS research “will focus on the evaluation of novel biomarkers including microRNA for ALS, and the development of data-driven prediction models that provide individual patient forecasts of ALS progression,” Fergal O’Brien, PhD, RCSI’s deputy vice chancellor for research and innovation, said in a separate press release.

Of note, changes in specific microRNAs, small molecules that suppress the activity of other genes, have been associated with ALS.

FutureNeuro research has been focused on “better data-driven approaches that will provide the right [therapy] for the right patient at the right time,” said David Henshall, PhD, FutureNeuro’s director and professor of molecular physiology and neuroscience at RCSI.

“By working with ADAPT, this unique collaboration between clinicians, computer and data scientists is ideally positioned to bring about change,” Henshall added.

The Ireland-based researchers will work together with Treatment Research Initiative to Cure ALS (TRICALS), an independent consortium of leading ALS experts, patients, and patient advocacy groups across Europe dedicated to finding a treatment for ALS. National and international industry partners and charities, including patient organizations, will also take part in the project.

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Precision ALS will also provide an interactive platform for clinical research in ALS across Europe that will use AI to analyze large amounts of data gathered in a timely and cost-effective manner from multiple international sites.

By building the largest, international, multimodal dataset aimed at precision medicine for ALS, the researchers hope to develop new and effective therapies for subpopulations of ALS patients.

Leo Varadkar, Ireland’s minister for enterprise, trade, and employment, and who launched the project, said that Precision ALS “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.”