On 5 March 2025, we hosted a Translational Science Summit on MND and Rare Dementias. From leading researchers, to venture capital specialists, to people living with MND, we had 35 speakers deliver 12 sessions throughout the day. Bringing together different disciplines allowed us to spotlight vast perspectives of the research landscape and learn from each other as we focused on our collective goal: improving outcomes for people with MND and rare dementias.
Despite these diverse perspectives, commonalities and key themes arose between sessions. Below are some of the significant takeaways from the day:
Learning from Cancer Research: addressing heterogeneity in dementia
Throughout the summit, many parallels were drawn between where cancer research was 30 years ago, and where dementia is today.
Dementia is an umbrella term describes a collection of disorders that share some features, but this is a very crude description; no one would think of cancer like this now.”
Siddharthan Chandran, MacDonald Professor of Neurology & Director, UK Dementia Research Institute
We are increasingly aware that different types of rare dementias, such as frontotemporal dementia, involve different underlying pathologies, and patients want to understand more about the molecular basis of their disease. Larger data sets and initiatives such as Our Future Health can improve our understanding of heterogeneity.
Speakers emphasised that valuable learnings can be taken from how the cancer research landscape has addressed this challenge and begun to develop more targeted treatments. These achievements can serve as a benchmark for what is possible in dementia.
Recognising the pathological complexity of dementia and improving diagnostic accuracy will improve the recruitment and stratification of patients for clinical trials, allow more accurate reporting on the effectiveness of drugs and help us to move towards precision treatments. As one of our speakers, Jenna Gregory aptly said, “We didn’t get better at treating cancer; we got better at diagnosing it.”
Reducing the time to diagnosis
A key priority identified by all speakers was the need for earlier diagnoses, alongside a strong call for less invasive diagnostic methods. Early and accurate diagnosis empowers patients with information that can guide symptom management, longer-term health planning and clinical trial participation. Late diagnosis further complicates recruiting patients with rare dementias for clinical trials as it can render the already limited patient population ineligible.
A multimodal diagnostic approach that combines fluid biomarkers, genetic testing, imaging, speech recognition patterns, and digital biomarkers could lead to faster and more accurate diagnoses. However, biomarker research is not without its challenges. It was emphasised that data sharing and thorough dissemination of research are critical to preventing duplication of efforts and ensuring collaboration opportunities are not missed. Larger datasets will enhance the power of neurodegenerative biomarker studies and play a vital role in establishing standardised diagnostic criteria.
Keep it collaborative, keep it coordinated and keep communicating about it. Please keep working together.”
David Setters, Patient representative at United 2 End MND
Improving clinical trials: quality, accessibility, and patient-centric design
Speakers, including our expert panel of people living with MND, stressed the need to improve the quality of clinical trials to expedite the delivery of treatments to patients. A key focus is generating robust preclinical evidence to ensure drug candidates can enter trials with greater certainty and a higher likelihood of success. Drug repurposing was presented as a valuable strategy to reduce both the time and costs of treatment development, with panellists emphasising the need to challenge the perception that repurposed drugs are inferior to new compounds. This year, we aim to fund and support 4 preclinical-stage MND drug repurposing projects to generate robust data and advance candidates to later-stage trials.
The use of biomarkers to better recruit, stratify, and identify responders is also paramount to improving trial design. Our venture capital specialists stressed that this approach would enhance drug candidates’ chances of success and, therefore, the likelihood of capturing the interest of large pharmaceutical companies.
Consulting people with lived experience when designing trials will ensure they align with what patients deem to be ‘clinically meaningful’ and account for their preferences on treatment administration methods, dosing schedules, and adverse event profiles.
Beyond improving trial design, it is essential to democratise and decentralise clinical trials to make them more accessible, particularly for patients with rare and ultra-rare dementias, where populations are widely dispersed. The rare dementia field has been praised for its approach to overcoming these constraints, including forming collaborative networks, factoring in travel costs for patients, and taking a global approach to deliver trials across borders. This model offers valuable lessons for trials in more common conditions.
With these reflections in mind, our Chair of Neurodegeneration, Roger Barker, left us with an important and optimistic reminder:
If a trial doesn’t give you a positive outcome, that does not mean it is a failed trial. It is important to remember that there are lots of lessons that can be learned from each clinical trial.”
Roger Barker, Chair of Neurodegeneration, LifeArc and Professor of Clinical Neuroscience, University of Cambridge
“We go further, and we move faster when we work together.” – Sam Barrell, CEO, LifeArc
While the summit highlighted actionable ways to improve the research landscape for MND and rare dementias, it also provided an opportunity to recognise how far the field has come.
We now understand many key pathways involved in MND, such as oxidative stress, mitochondrial dysfunction and protein misfolding. Many of these mechanisms occur in other neurodegenerative diseases and rare dementias, presenting a huge opportunity to learn from each other. With a clear grasp of the principal pathways in MND, the next step is to determine the sequence of these events and identify the most therapeutically targetable ones. An effective treatment for one neurodegenerative condition could potentially benefit many others.
Global collaboration has played a transformative role in enhancing recruitment for clinical trials and ensuring that more diverse data is collected and shared. Our understanding of brain health has improved and there are now 14 recognised risk factors for dementia, fuelling a positive shift towards thinking about prevention. Increased awareness of MND and rare dementias has led to more people seeking diagnoses and participating in clinical trials. As awareness grows, so too does funding, all of which helps us to turn the dial and encourage people to think of dementias as treatable conditions.
“There is no silver bullet, but that doesn’t stop us looking.”
Jill Douglas, sports broadcaster and patron of My Name’5 Doddie Foundation
These are just a few key themes that emerged from what was a jam-packed day of rich, informative and honest discussions. If you missed any of the sessions or want to revisit them, they will be available to watch online soon. Finally, a big thank you to all of our amazing speakers and attendees for making the day such a success.
