Patients at the Heart of Research: IMP’s Role in the SIMPATHIC Drug Repurposing Project
The SIMPATHIC project continues to make important progress in advancing drug repurposing for rare neurological, neurometabolic, and neuromuscular disorders. As highlighted in the consortium’s latest newsletter , the initiative brings together cutting-edge computational science, innovative clinical trial approaches, and strong patient involvement to accelerate access to potential therapies. IMP is proud to be an active partner alongside other patient organisations in this ambitious Horizon Europe–funded programme, contributing across multiple work packages and ensuring the patient voice remains at the heart of the project.
A key area of IMP’s involvement is in shaping the clinical direction of the project. Jo de Bry, IMP’s Director of Development and Operations, sits on the Clinical Trial Steering Committee, ensuring that trial design is fully informed by patient needs and real-life considerations. This includes significant work on the potential clinical trial of Sildenafil for patients with Leigh Syndrome, a trial with meaningful potential for families affected by this severe mitochondrial disorder. Jo provides guidance on visit schedules, assessments, drug delivery and patient recruitment strategies, helping to ensure that the trial is developed with patients and families firmly in mind. Additionally, the submission of an in-depth Scientific Advice request to the European Medicines Agency (EMA), with a response expected between January and February 2026, marks a crucial milestone in validating SIMPATHIC’s planned clinical trial design.
Jo gave a presentation on the importance of patient feedback in clinical trial design and patient recruitment strategies at the SIMPATHIC Annual Meeting on 1 December. She reinforced how essential it is to co-develop trial materials and recruitment pathways with patients to improve clarity, reduce burden and increase meaningful participation.
IMP Chair, Paula Morandi plays an equally important role, representing the mitochondrial disease community on the SIMPATHIC Multi-Stakeholder Advisory Board. Her participation ensures that patient organisations have a strong influence on the project’s strategic priorities and that the lived experiences of those with rare diseases are embedded across SIMPATHIC’s work.
Beyond governance, IMP is working with other partners on capacity-building activities that will have long-term benefits for patient communities. A major initiative is the training programme for patient advocates on drug repurposing, developed collaboratively and to be delivered through EUPATI. This programme will provide patient advocates with accessible, structured knowledge on drug repurposing—from the underlying science to regulatory processes—empowering them to engage more effectively in research, policy development and clinical trial planning.
The scientific foundation of SIMPATHIC’s drug repurposing efforts is also highlighted in the latest newsletter. The project makes extensive use of knowledge graphs, which organise information about diseases, genes, proteins and drugs into interconnected networks. These networks can then be analysed using Graph Neural Networks (GNNs)—a form of Artificial Intelligence that learns from how different pieces of data are linked. In simple terms, GNNs examine a huge web of connections and can spot patterns and potential treatment opportunities that humans might not easily see. This helps researchers identify existing medicines that could potentially be repurposed more quickly and efficiently for rare disorders. A recent publication in Computers in Biology and Medicine explains how this approach provides an innovative foundation for discovering new therapeutic options.
Clinical innovation is another essential element of SIMPATHIC’s strategy. The consortium recently presented its novel basket trial design at ICIEM 2025 (International Congress of Inborn Errors of Metabolism). This innovative model allows multiple rare disease indications to be evaluated within a single trial structure, increasing efficiency and potentially speeding up patient access to promising therapies.
Other significant updates across the project include the harmonisation of multiple semantic knowledge graphs into a single comprehensive resource, and important enhancements to the Biovista Vizit platform. The Biovista Vizit platform has also been enhanced with new data sources, including clinicaltrials.gov, allowing researchers to visually explore clinical trial information alongside scientific evidence. These updates—building on earlier additions such as Human Phenotype Ontology and Protein Data Bank data—make Vizit a more powerful tool for identifying promising drug repurposing opportunities.
As SIMPATHIC prepares for its next consortium meeting in mid-2026, IMP remains committed to ensuring that the perspectives of patients and their families drive the project’s direction and outcomes. By contributing to clinical steering, participating in the development of patient advocacy training, offering strategic guidance and presenting on patient-centred approaches, IMP—working alongside fellow patient organisations—continues to strengthen the impact and relevance of this important European initiative for the rare disease community.