Advancing brain research and treatment

FutureNeuro, now entering its second phase, aims to transform the healthcare experience
for patients with neurological conditions

Brain disease is one of the biggest challenges for public health, society and the economy in Ireland. With one-in-three individuals affected over their lifetime, the annual burden on healthcare and welfare systems amounts to an estimated €30 billion. In Ireland alone, more than 800,000 people have conditions impacting the brain and central nervous system, such as epilepsy and motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS) to Parkinson’s disease, multiple sclerosis (MS), and acquired brain injury. Compounding these challenges, over 30 per cent of people affected also have psychiatric disorders, adding complexity to their care.

FutureNeuro is a Science Foundation Ireland (SFI) interdisciplinary research centre, hosted by the RCSI. It is committed to alleviating this burden by accelerating progress and translating innovations into real-world solutions. With a focus on improving diagnosis, developing targeted treatments, and advancing healthcare management, the centre is committed to making tangible differences in patient outcomes. Since it launched in 2017, FutureNeuro’s research has led to transformative interventions, including genetic testing to identify patients most suitable for life-changing therapies. The centre recently launched its second phrase, supported with a €17.9 million Government investment through SFI.

Navigating the path forward: FutureNeuro’s vision for the future              

As FutureNeuro embarks on its second phase, and fuelled by notable achievements to date, its vision for the future is to change the patient journey. However, the reality is that most brain diseases still lack effective treatments and diagnosis remains a prolonged process for many. Recognising the scale of the challenge ahead, the centre has brought together the very best scientists, clinicians, and academic and industry partners, working together on an integrated research programme to progress the centre’s journey to being a world-leading research institute that directly connects with the needs of patients and their clinicians.

Expanding the team

To realise transformative advances, FutureNeuro, led by Prof David Henshall, Centre Director and Professor of Molecular Physiology and Neuroscience, RCSI, is scaling up its operations and integrating new science, talent, and expertise in brain research from prestigious institutions across Ireland. In addition to the RCSI, these include: Trinity College Dublin; Dublin City University; University College Dublin; University of Galway; University College Cork; Maynooth University; and South East Technological University. Most importantly, the centre is integrated with a research-engaged national clinical network which collectively informs the research questions and ensures valuable access to patients and their data in order to make the research relevant and translational.

While maintaining a strong focus on epilepsy and MND, FutureNeuro 2 is broadening its impact to include neurodevelopmental disorders, MS, Parkinson’s disease, traumatic brain injury, and their intersections with psychosis and mental health. FutureNeuro 2 has grown its network to include over 30 research leaders, their dedicated teams and new enabling infrastructure. This collective expertise will further our understanding of the mechanism of action of the brain and brain disease, enable the identification of new molecular diagnostics, neurogenetics, noncoding RNA biology, and gene therapies.

Moreover, FutureNeuro 2 recognises the invaluable potential in cross-pollinating discoveries across different disease areas. By leveraging insights gleaned from one condition to inform research and treatments for others, the centre aims to maximise impact and efficiency, ultimately accelerating progress towards addressing the broader spectrum of brain diseases.

New research frontiers: From diagnostics to therapy and beyond

FutureNeuro’s research is built on three connected themes – diagnosis, therapy, and digital health. The centre is rolling out an integrated programme of work across the eight academic partners which will be complemented by collaborative projects with world-leading industry partners with shared research objectives.

Diagnosis

This strand of the programme concentrates on discovering genes that cause brain disease or influence how people respond to treatments. The diagnostics team is examining the chemistry of the blood and fluid that surrounds the brain to look for answers to causes and prognosis, recognising that errors in DNA may not always be the sole cause.

By analysing the chemistry outside brain cells, researchers can create a molecular fingerprint or profile specific to a particular brain condition. When combined with brain imaging, artificial intelligence (AI), and recordings of brain activity, these biomarkers enable increasingly accurate predictions of how a brain disease will develop in an individual over time.

Genomics is transforming the diagnosis of neurological diseases, paving the way for the emergence of precision medicine. FutureNeuro has been instrumental in incorporating genomics into clinical practice, leading to faster and more accurate diagnoses. Currently, Prof Gianpiero Cavalleri, the Deputy Director of FutureNeuro, is co-leading an Irish consortium in contributing to the €20 million Digital Europe Call, ‘Federated European Infrastructure for Genomics Data’. This initiative aims to advance the European 1+ Million Genomes Initiative by creating a unified platform for genomic data across Europe.

Therapeutics

FutureNeuro’s therapeutics research is organised as a pipeline, drawing on the insights from our diagnostics teams to identify potential targets for future medications. A groundbreaking capability lies in our ability to generate human brain cells in the lab, allowing us to screen for safer and more effective drugs. But, to fully capitalise on the promise of genetics requires us to use gene therapy. Encouragingly, remarkable progress has been achieved. Clinical investigator Prof Orla Hardiman is trialling a number of pioneering gene therapy-based treatments for MND in collaboration with world-leading pharmaceutical companies.

The centre is also looking at how to safely deliver genes to precise areas of the brain, to control their activation via on/off switches and minimise potential side-effects. Research extends to areas such as small non-coding RNAs and the biology of the blood-brain barrier. With a sharpened focus on accelerating clinical development and understanding the intricacies of the human brain, FutureNeuro 2 is charting a course towards neurotherapeutics of the future.

Digital health

In the rapidly evolving landscape of digital health, innovations are fundamentally reshaping healthcare delivery, access, and system operations. FutureNeuro’s research programme stands at the forefront, driven by the insights of clinicians and patients alike. Leveraging a rich data environment and cutting-edge digital technologies, they are co-designing solutions for proactive, personalised, and cost-effective healthcare.

The centre is laying the groundwork for a learning health system. This involves ethically integrating diverse datasets, including electronic health records, and passive data from connected devices in homes. Furthermore, responsible AI solutions aim to extract valuable insights from data, aiding clinical decision-making, and continually improving patient care.

In tandem, FutureNeuro 2 is pioneering a smartphone app-based digital therapeutic. Tailored to capture lifestyle data, it provides personalised interventions for mental wellbeing. Moreover, through strategic industry partnerships nurtured by programmess like SFI SPOKE (EMPOWER and Precision ALS), they are advancing next-phase projects focused on health data analytics and building trust in the secondary use of data, alongside the utilisation and adoption of technologies such as AI. 

Mental health

Mental health research is threaded throughout the new programme. In diagnostics, a significant new study is underway to look for changes in blood chemistry, aiming to identify people at risk earlier than is currently possible. In the drug development programme, researchers are targeting genes associated with psychosis while incorporating assessments for depression, anxiety, and psychosis across all drug development projects. This approach not only enhances drug efficacy, but also provides insights into addressing the underlying mechanisms of mental health disorders. In the digital therapeutics programme, efforts are focused on leading advancements in technologies that not only monitor, but also positively intervene in brain health. This includes digital therapeutics that offer individuals greater control and continuity of care.

Rare diseases

The Irish Government is committed to addressing rare diseases, with plans underway to create a national rare disease plan and a national genetics and genomics medicine network. FutureNeuro clinical researchers are developing patient registers for rare genomic diseases in order to stratify patients for clinical trials and be ready to implement advances in genomic medicine. Additionally, promising treatments are emerging at pre-clinical stage for epilepsy, including those for conditions like Dravet syndrome. FutureNeuro 2 will prioritise these often-overlooked conditions, aiming to drive progress and improve patient outcomes.

New research projects

FutureNeuro 2 is already working on some remarkable projects that are taking a deep dive into the workings of the human brain. One such project involves recording patterns of electrical activity of brain cells, essentially eavesdropping on neurons and listening for when they misfire. This is possible because sometimes doctors will implant electrodes into the brains of people with treatment-resistant epilepsy to pinpoint the source of their seizures. The researchers have found a way to measure gene activity at the site of those recordings. This is helping to discover new genes that influence brain function and could hold promise for clinical decision-making. Like so much of the centre’s work, this research relies on specialised scientific skills and collaboration with their dedicated clinical partners. This is only made possible by the robust framework upon which FutureNeuro 2 is built.

Another exciting study aims to understand how to repair leaky blood vessels in the brain. In a healthy brain, blood vessels are tightly sealed, but in many neurological diseases, they become permeable. This hallmark is also associated with psychosis. By reapplying the genetic ‘glue’ responsible for maintaining the blood-brain barrier, researchers hope to open the doors to a completely new type of treatment.

Education, public, and patient engagement

Another pivotal evolution within FutureNeuro 2 is the deeper integration of patients within the centre. They contribute to identifying research priorities, evaluating progress, and aid in communication with the public, funders, and policymakers, ensuring that research aligns closely with patient needs. Our patient panel played a key role in preparing the research strategy for phase 2. The centre is now actively developing disease-specific patient panels to be firmly embedded across all areas of their research into other brain diseases, including Parkinson’s and MS.

Education and public engagement are other key components of their mission. The Brain Health webinar series, in partnership with Epilepsy Ireland, is just one example of the strategic collaborations they have undertaken to help break down barriers and make epilepsy research accessible to all. The team will also raise awareness of brain health and wellness with the wider community through an innovative education and engagement campaign called My Moving Brain, co-delivered across Ireland with patient advocacy groups and local sports partnerships as part of an SFI Discover Award. This initiative will build on their highly successful Cell Explorers school programme that will continue to inspire the next generation of life scientists. 

With over 400 engagement activities undertaken, including collaborative events with charitable organisations, enlightening workshops in schools, thought-provoking discussions at Pint of Science gatherings, the creation of informative blogs for wider audiences, and valuable contributions to national policy, the centre will continue to raise awareness and capture the public’s imagination, supported by a dedicated education and public and patient engagement lead.

Industry 

Collaborating with industry is fundamental to the success of FutureNeuro. To date, the leadership team have undertaken more than 30 collaborative research projects, including partnerships with Roche, UCB, IQVIA, Novartis, Janssen, and Congenica. These include bringing new diagnostic supports to market, advancing a pipeline of new drugs through development and testing, and expanding digital health solutions to enable patients and their clinicians to co-monitor their health better than ever before. In phase 2, the team will expand engagement with industry with a collective aim to advance new therapeutics and precision medicines, identify faster and more precise diagnostic tools, leverage the power of AI to accelerate discoveries based on clinical data, and promote Ireland as a preferred location for early-stage clinical trials.

As FutureNeuro 2 embarks on its mission to revolutionise brain research and treatment in Ireland, it commits not only to unravelling the complexities of neurological conditions, but also to leading the charge in translating these findings across various brain diseases, paving the way for improved patient outcomes and transformative strides in healthcare.

For more information on the work of FutureNeuro go to www.futureneurocentre.ie