A new European project kicked-off on 10 – 11 July in Amsterdam, The Netherlands namely SIMPATHIC, which stands for Accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders by exploiting SIMilarities in clinical and molecular PATHology, at the premises of project coordinator Radbound University Medical Center (UMC).
SIMPATHIC project partners gathered to meet each other for the first time, meet consortium members and discuss their role and methodologies in the project.
The project is funded by the European Commission’s Horizon Europe Program on tackling diseases. SIMPATHIC is a five-year project, with a main objective to accelerate drug repurposing for rare neurological, neurometabolic and neuromuscular disorders. SIMPATHIC’s main accelerating innovation is the simultaneous drug development for groups of patients with different genetic diagnoses but overlapping neurological symptoms and molecular pathomechanisms. The consortium comprises patients & families, a patient training organization, pharma companies, basic, translational and clinical scientists, bio-informaticians, ethicists and methodologists.
The focus on Drug repurposing is of significant importance, hence it can fill an important gap for rare disease patient groups with large unmet medical needs. In comparison to traditional drug development, drug repurposing increases time and cost efficiency for drug development.
SIMPATHIC’s key outputs of accelerating the drug repurposing pathway will include:
• Standard operating procedures for culturing stem cell-derived neuronal cell models with proven relevance for clinical symptoms and amenable to high-throughput drug screens.
• New drug repurposing candidates with proven efficacy in advanced brain-on-a-chip and 3D brain organoid models, as demonstrated by reversal of molecular biomarker signatures and cellular readouts associated with clinical symptoms
• Designs of innovative basket clinical trials to which patients with different disorders are recruited, utilizing and aggregating personalized clinical endpoints.
• A training module for patients and patient organizations to empower them as drivers of the drug repurposing pathway.
• Blueprints for intellectual property strategies, business models, regulatory dossiers and patient access strategies, developed in co-developed between all relevant stakeholders.
Stay tuned to www.simpathic.eu for further information, news and events.