Motor neuron disease

Research collaboration to explore precision treatments for motor neuron disease

  • International
  • Research

Motor neuron disease (MND), also known as amyotrophic lateral sclerosis (ALS) is a condition that affects hundreds of people in Ireland today. Approximately 140 people in Ireland are diagnosed with MND every year and worldwide there are approximately 140,000 new cases diagnosed annually.

There are no effective treatments for MND and it can affect people in different ways. The disease can cause progressive decline in movement, cognition and behaviour and life expectancy can vary from three months to many years from diagnosis.

Research has shown that the disease is caused by variable combinations of faulty genes that likely interact with lifestyle and environment. As the disease has different causes, different patterns of progression and can affect people in different ways, large amounts of data is required to understand these differences and inform clinicians on treatments.

A new €10 million research programme called Precision ALS has been developed by two Science Foundation Ireland (SFI) Research Centres, ADAPT and FutureNeuro, the RCSI-based centre for chronic and rare neurological diseases. The researchers will work in partnership with TRICALS, an independent group of leading ALS clinician scientists, patients and patient groups from across Europe, as well as national and international industry partners, charities and patient organisations.

Personalised treatments for individual patients

Precision ALS is a programme based on the precision medicine approach. Precision medicine, combines the various genetic factors and functional defects that underpin disease presentation and progression, with environmental and lifestyle factors, in order to personalise and tailor the treatment response to each individual patient.

The Precision ALS programme will combine clinical research with cutting-edge data science and artificial intelligence (AI) to analyse large amounts of data relating to MND to understand the disease and how to treat it. The findings from this data will be used to develop new treatments for MND and to offer potential benefits for other rare conditions and diseases.

At RCSI, the Precision ALS research led by FutureNeuro principal investigator Professor Jochen Prehn, will focus on the evaluation of novel biomarkers that circulate in the blood of ALS patients including non-coding RNAs. The team will develop data-driven prediction models that incorporate differences in these biomarkers between patients, as well as functional and clinical data to provide individual patient forecasts of ALS progression.

The programme will address the issues with gathering new data at scale in a timely and cost-effective manner increasing understanding amongst scientists of MND and how to treat it.

AI is key to identifying contributing factors and increasing the power of scientists to discover changes linked to disease. The work from Precision ALS will lead to transformational change for patients with a ripple effect that will positively impact society. The programme will break boundaries and develop targeted therapies that will enable the right drug to be provided for the right patient at the right time.

The collaboration will directly benefit healthcare and patient communities, reduce the economic cost of these diseases and yield new knowledge, approaches and treatments with the potential to improve the lives of many.

RCSI is committed to achieving a better and more sustainable future through the UN Sustainable Development Goals.