For people living with rare neuromuscular disorders, every new scientific discovery means renewed hope. These conditions, though individually uncommon, affect thousands of families across Europe. They are progressive, complex and often without effective treatment.
The European Union–funded DREAMS project — Drug Repurposing with Artificial Intelligence for Muscular Disorders — is changing the way we approach this challenge.
Instead of developing one therapy for one disease at a time, DREAMS is exploring how artificial intelligence (AI) can uncover connections between several rare disorders that share similar biological mechanisms. Its goal is simple yet revolutionary: to build a digital platform that finds treatments capable of addressing multiple diseases simultaneously.
A new mindset for rare disease research
Traditional drug discovery often follows a narrow path — one molecule, one target, one disease. For rare conditions, this approach is slow, expensive and limited by small patient populations. DREAMS offers a new perspective: by focusing on what these diseases have in common rather than what separates them, it aims to multiply the impact of each discovery.
The project brings together nine organisations from six European countries, combining expertise in stem-cell biology, pharmacology, data science and clinical research. Led by the CECS/I-Stem Institute (Centre d’Études des Cellules Souches), the consortium is united by a shared mission — to accelerate new treatments for people affected by rare neuromuscular disorders.
From living cells to intelligent data
At the heart of DREAMS lies the creation of specialised cellular assays — laboratory tests built on skeletal muscle cells derived from induced pluripotent stem cells (iPSCs).
These cells behave like real muscle tissue and make it possible to study how disease develops at a cellular level. By testing a range of compounds and observing how these cells respond, researchers can gather valuable biological data that feeds directly into DREAMS’ AI models.
This data-driven approach allows scientists to identify common drug targets across different diseases. Rather than treating each condition in isolation, DREAMS builds a shared understanding of the cellular and molecular disruptions that cause them — such as autophagy dysfunction or desmin disorganisation, both known features of many neuromuscular disorders.
The power of AI models for drug discovery
The DREAMS digital platform integrates these vast datasets using advanced AI and machine-learning algorithms developed by the project partner Kantify.
By analysing patterns across different diseases, the system can predict which biological targets are most promising and which existing compounds might be repurposed safely for new uses.
AI’s role here is not to replace scientists but to amplify human insight. It helps researchers make sense of enormous, complex data and focus on the most promising directions.
The result is a faster, more precise discovery process that reduces both costs and risks — key advantages in the field of rare diseases, where resources and time are precious.
Repurposing and beyond
DREAMS focuses first on identifying and validating safe, already-approved drugs that might benefit more than one disease. This process, known as drug repurposing, can dramatically shorten the path from discovery to patient application because the safety profile of these medicines is already known.
However, the ambition of DREAMS goes further. By revealing new shared mechanisms, the platform can also point to novel molecules or new combinations of treatments, opening the door to therapies that may never have been considered for these conditions before.
The project’s five target diseases — Dynamin 2 Centronuclear Myopathy, Duchenne Muscular Dystrophy, Emery-Dreifuss Muscular Dystrophy, Pompe Disease and Danon Disease — provide the starting point for this exploration. Each of them has known genetic origins and molecular markers, making them ideal test cases for this multi-disease methodology.
Towards smarter, adaptive clinical trials
Even when promising therapies are identified, clinical trials in rare diseases face unique challenges: small patient numbers, high variability, and ethical constraints.
To address this, DREAMS is designing adaptive clinical trial protocols, developed with input from patients and regulatory experts.
These innovative models aim to make trials more flexible and inclusive — adapting as new data emerge and allowing researchers to test multi-disease hypotheses within realistic timeframes.
This is not only about scientific progress but also about respect for the people involved. DREAMS recognises that patients are partners in research, and that their experience is essential in shaping studies that are both effective and humane.
A reusable platform for future research
One of the most exciting aspects of DREAMS is its reusability. The combination of AI models, standardised cell assays and data protocols is designed to serve beyond this project.
Once validated, the DREAMS platform will become a blueprint for future rare-disease research, allowing other teams to apply the same principles to different conditions.
This makes DREAMS more than a single project — it’s a model for how Europe can scale innovation in personalised and precision medicine.
By connecting disciplines, data and communities, DREAMS builds the foundation for a new era of biomedical research that is collaborative, transparent and impact-driven.
The European impact
DREAMS is funded under the Horizon Europe programme — a reflection of the EU’s commitment to harnessing cutting-edge science for societal good.
Its outcomes promise not only new therapeutic candidates but also a tangible demonstration of responsible, human-centred AI applied to healthcare.
The project’s expected benefits reach far beyond the lab:
- Scientific – generating validated evidence on the integration of AI and iPSC-based screening.
- Societal – improving quality of life for patients living with rare neuromuscular disorders.
- Socio-economic – reducing the cost of developing therapies by identifying shared mechanisms.
- Marketability – creating valuable knowledge and tools that can be leveraged by future European research initiatives.
A glimpse of the future
As DREAMS advances, it is shaping a future where AI and biology work hand in hand to tackle the toughest medical challenges.
Imagine a world where data from one rare disease can help cure another; where each discovery accelerates the next; where knowledge circulates instead of staying locked in silos.
That is the world DREAMS is working to build — one where innovation travels faster, collaboration runs deeper, and patients no longer have to wait a lifetime for a breakthrough.
Discover more about DREAMS at dreamshorizon.eu
Follow the project on X and LinkedIn for the latest updates and results.
