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The Longitude Prize on ALS: Real World Implications

Today, the Longitude Prize on ALS was launched, an initiative that aims to incentivise the use of AI to transform drug discovery for the treatment of ALS. What is the true impact of the Prize? We spoke to Tris Dyson (Founder & Managing Director, Challenge Works) and Amalia Papanikolaou (Data Lead, Challenge Works) to find out more about the initiative and the real world implications for patients.

Understanding ALS

To understand the importance of the Prize, we must first understand amyotrophic lateral sclerosis (ALS) itself. ALS is the most common form of motor neurone disease (MND) and gradually prevents messages from motor neurones from reaching the body’s muscles. This causes the muscles to weaken, stiffen and waste, which affects a person’s ability to walk, talk, eat, drink and breathe. Some also experience changes to thinking and behaviour. The disease affects everyone differently; not all symptoms will affect everyone, or in the same order. Rates of progression also vary person to person, making the course of the disease difficult to predict.

Despite its profound impact on people’s lives, there is currently no cure for ALS. However, some limited treatments exist that can help manage symptoms or modestly slow disease progression. These include Riluzole, the first drug approved for ALS, that can extend survival by a few months; Tofersen, a newer treatment, designed specifically for a small subset of ALS individuals with a mutation in the SOD1 gene; highly-specialized clinics, typically involving a specialist nurse and occupational therapy to help make everyday tasks easier; and physiotherapy to maintain strength and reduce stiffness.

What is the Longitude Prize on ALS?

The Longitude Prize on ALS is an international, £7.5 million challenge prize toincentivize the use of AI-based approaches to transform drug discovery for the treatment of ALS.

It is designed and delivered by Challenge Works, supported by Nesta, the global challenge Prize expert; and is principally funded by MND Association. Additional funders include Nesta, the Alan Davidson Foundation, My Name’5 Doddie Foundation, LifeArc, FightMND, The 10,000 Brains Project, Answer ALS and The Packard Center at Johns Hopkins University.

The Prize will award a total of £7.5 million over multiple stages to fund teams of researchers that will be working closely alongside tech companies to use AI to identify the most promising drug targets for future treatments for ALS.  

To enable this research, the Prize has brought together global charities and organizations to begin uniting multiple sources of data, helping to build one of the most comprehensive ALS datasets of its kind. In the past, ALS data has often been scattered across institutions, each with its own access limitations and formats. The Longitude Prize on ALS aims to make this data more accessible by bringing it together in a single, trusted environment.

This approach, combined with rapid advancement of artificial intelligence (AI), makes this the perfect time to support biomedical researchers to work with AI tech firms to use this data to identify therapeutic targets  that could support the development of new ALStreatments. 

Why is now the time to be launching the Longitude Prize on ALS?

Medical professionals have been working to find an effective treatment for ALS for decades. There are a number of reasons for the slower pace of progress, including genetic complexity, poor clinical trial design and diagnostic delays – amongst others.

AI for drug discovery is a growing area of interest that has yielded significant results with other diseases and has gained increased support in recent years. For example, AI has been used to identify compounds that have aided the understanding of carcinogenesis. Subsequently, treatments for various cancers have been developed. This was achieved through machine learning and led by scientists at Harvard Medical School.

Given that there is a vast amount of previously untapped patient data on ALS, the team behind the Longitude Prize on ALS has identified the application of AI to untangle complex data sets as a potential means to identify a cure for the treatment of ALS.

Huge advances in AI capabilities in recent years, combined with the recent advances in understanding the complexity of the disease; as well as better availability of patient data, present innovative approaches to drug discovery as a gamechanger. It is for this reason that the time is ripe for launching the Longitude Prize on ALS.

By matching leading ALS scientists with AI and biotech experts, and the ALS community, combined with essential funding and unprecedented data access, the Prize will supercharge discovery.

How exactly could AI be used to facilitate research in this space?

AI has the potential to transform drug discovery by enabling research to rapidly analyse data, including multiple datasets and types of datasets at once, streamlining the time-consuming process of drug discovery.

AI presents a particularly huge opportunity for heterogenic diseases, such as ALS. When a disease is heterogenic, it manifests in diverse ways, including variability in its causes, clinical presentation, progression, and response to treatment. This lack of consistency complicates research.

Researchers need more data for heterogenic diseases than diseases that have a singular cause (such as a point mutation in sickle cell anaemia). The more data there is, the higher the likelihood of picking up meaningful signals among the noise in the dataset – which can lead to discovery.

AI approaches are becoming more advanced and at the same time, we have access to more ALS data than we ever had before. AI can help us make sense of complex data and traits within the data, allowing us to understand the disease better. This means that the opportunity for AI to transform drug discovery in ALS is huge.

However, processing this data manually would take years. AI’s capacity to process huge amounts of biological data is a gamechanger for developing a treatment for a disease that has a high rate of clinical failure. This high failure rate in ALS clinical trials is often attributed to the disease’s unclear etiology and pathophysiology. AI will change this.

What is the desired outcome of the Longitude Prize on ALS?

Ultimately, the desired outcome of the Prize is to identify new treatment pathways that could lead to the development of a new drug to treat – or even cure – those living with ALS. Given the significant genetic and mechanistic overlap between ALS and neurodegenerative diseases, in particular frontotemporal dementia (FTD), it is possible that future treatments could be applicable to those living with other neurodegenerative diseases.

Beyond the ultimate goal of discovering a potential therapeutic target, the hope is that the Prize will raise international awareness of the disease and draw attention to the urgent need for continued research and increased funding. In 2022, the UK government pledged £50 million to support MND research over five years; comparatively, it spends £112 million annually on cancer research and has pledged £160 million per year for dementia research.

The Longitude Prize on ALS is being run by Challenge Works – a leader in the design and delivery of high-impact challenge prizes that incentivise cutting-edge innovation for social good. Challenge Works has funded numerous successful challenges. To date it has worked on over 100 challenge prizes; announced over 300 winners and over 800 finalists; and distributed over £260 million to innovators and participating teams. 

How can I get involved?

To find out more about the Longitude Prize on ALS, including how to apply, head to the website: https://als.longitudeprize.org/