“One patient can change everything”
A conversation with experts Patrik Verstreken and Albena Jordanova on new horizons in neurodegeneration
Neurodegenerative diseases like Alzheimer’s, ALS, and Parkinson’s each come with distinct symptoms and, in many cases, multiple underlying causes. Yet researchers are finding common ground in the ways these conditions are investigated and in the approaches being developed to treat them. At the New Horizons in Neurodegeneration conference, scientists from across the globe will explore these shared mechanisms to push the field toward smarter, faster, and more precise treatments.
This year's event also serves as a celebration, marking the 10th anniversary of VIB Conferences. The focus of the conference has broadened significantly over the years, evolving from a primary emphasis on Alzheimer’s disease to a more comprehensive exploration of neurodegeneration, including ALS, frontotemporal dementia, Parkinson’s disease, and peripheral neuropathies, reflecting the diverse research landscape at VIB today.
We spoke with organizing committee members Prof. Patrik Verstreken (VIB-KU Leuven Center for Brain & Disease Research) and Prof. Albena Jordanova (VIB-UAntwerp Center for Molecular Neurology) about the importance of research models, promising shifts in the field, and what gives them that “aha” feeling.
Hi both, thanks for taking the time. Could you introduce yourself and what you do in your own words?
Patrik: My lab studies how synapses, or brain connections, deteriorate in neurodegenerative diseases like Parkinson’s and Alzheimer’s. Synaptic loss is one of the first issues to arise in these conditions. If we can prevent this loss, we might stop the cascade that later leads to neurodegeneration. And unlike neurons, synapses can regrow. Our ultimate goal is to understand how to restore that functionality.
Albena: My team starts by studying individual patients with hereditary neurodegenerative diseases of the peripheral nervous system. We then further recapitulate our genetic findings in different model organisms to test mechanistic hypotheses on why and how neurons are primarily affected, and gain a broader understanding of neurodegeneration in humans. Ultimately, we aim to return to the patient with enhanced insights and, hopefully, better treatment options.
"It’s a full circle: from patient, through model, back to patient."
On to the conference. Which speakers or sessions are you personally most excited about?
Albena: I’m most excited about the session on peripheral nerve degeneration. It’s an area that doesn’t get as much attention as the central nervous system, while peripheral neurons are essential for movement and overall body function. Peripheral nerve cells have unique morphological and functional characteristics, and I’m looking forward to learning from researchers who focus on that, like Stephan Zuchner and Charlotte Sumner.
Patrik: I’m especially looking forward to talks on fundamental disease mechanisms. The field is moving fast, and some of the first potential medications are making their way through clinical trials. I look forward to seeing this progress and what people are working on right now. There are many great speakers scheduled: Li Gan’s work on synaptic dysfunction and tau, Judith Steen’s research on protein isoforms and post-translational modifications, Felipe Opazo is doing incredible multiplex imaging… Lots to look forward to!
What model systems or technologies do you think are transforming how we study neurodegeneration?
Patrik: The newest technologies allow us to work with human tissue more. We can examine post-mortem samples with unprecedented detail using spatial technologies, perform an increasing number of biopsies on living tissue, and simulate the human brain with organoids. So we are really starting to work more and more with human material: this is the starting point for detecting what is going wrong in the disease. Of course, we still need animal models to show a direct link between cause and effect, but the fact that we can now directly investigate human brain material is a huge step forward.
Albena: I think we’ll see a combination of things. Firstly, we indeed need better, more humanized models. Second, it is essential to use a good mix of models, including computational ones, to fully understand the complexity of the nervous system. Additionally, we should include so far unexplored models, such as the research you are conducting with hamsters, Patrik. And (rare) patient cases will remain crucial: even a single patient can provide valuable insights into how the system operates. So, a focus on unique and clinically well-characterized patients, alongside a variety of humanized models, is the future of the field.
Where do you see the most promising shifts happening in neurodegeneration research?
Albena: Technology pushes the field forward and will continue to do so. Our responsibility as researchers is also increasing. There’s growing pressure from patient organizations and funding bodies for causal treatments. That pressure is an extra incentive to think harder about our models, our biomarkers, and how we measure success. This will also prepare the ground for industry and it emphasizes the role that academic scientists play in shaping these developments.
Patrik: We’re starting to see clinical translation. Fundamental discoveries are starting to prove their use in clinical settings, with human patients. It’s still early, but we’re finally catching up to fields like oncology. There are dozens of potential drugs in trials right now. I believe we’ll end up with combination therapies, cocktails tailored to different patient subtypes, just like in cancer. It’s an exciting time.
What are the biggest hurdles still slowing down the pipeline from discovery to therapy in neurodegeneration?
Patrik: Patient stratification is a huge issue. Right now, especially in big pharma, patients are often lumped into one group, which masks the effects of biomarkers and medications on subgroups. We need to stop treating these diseases as one-size-fits-all.
Albena: I agree. And academic researchers also need a better understanding of the drug development pipeline. We need to understand what the industry demands from us. VIB Discovery Sciences is great at helping bridge that gap, for example. We also need better biomarkers for early diagnosis so that any potential treatment can have a better chance to halt or reverse the neurodegenerative process in the patients.
On a personal note. You’ve both seen the field evolve over the years. What would you tell your younger self just starting out in neurodegenerative research?
Patrik: Take AI courses! And a lot of them.
Albena: It’s more general for me: Stay curious.
What part of your job do you find most satisfying?
Patrik: When someone walks into my office excited about a result, big or small. That moment when an experiment shifts your thinking or opens up a new direction is incredibly rewarding.
Albena: For me, it’s when I identify a genetic defect in a single patient and realize it reveals something new about biology. That one case can lead to fundamental insights, something that will change the textbooks, it’s an “aha” moment for both the patient and the science. It’s what rare diseases do best: they can turn everything upside down.
If there was one message or if you could explain one thing about your field to get across to policymakers, what would it be?
Patrik: Have some trust in researchers. Stop throwing so much administration at us.
Albena: I can only agree!
What’s a book, podcast, or film that you would recommend to everyone?
Albena: I would highly recommend “Time, Love, Memory”, a biography of biologist Seymour Benzer, written by Jonathan Weiner. All future scientists should read it. It was very inspirational for me.
Patrik: I would actually say the same! It’s a fantastic book about a fantastic scientist.
Thank you, both!
Want to join the experts? Register for the New Horizons in Neurodegeneration Conference on 9-10 October. The early bird deadline is 11 September!
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