Imagine finding a key that opens several different locks because the locks all share a similar internal mechanism. Instead of spending years making a new key for every single rare brain disease, this project looks for existing medicines that fit multiple diseases with similar biological patterns. They use tiny, lab-grown 'mini-brains' to test if these old drugs can fix the cellular damage before trying them in people.
Traditional drug development for rare diseases is too slow and expensive due to small patient populations. There is a critical need to find existing drugs that work across different genetic diagnoses with similar symptoms.
Standard operating procedures for neuronal cell models, 3D brain organoids for drug screening, and designs for basket clinical trials.
If you are a drug repurposing specialist dealing with the high cost of traditional drug development — this project developed blueprints for business models and regulatory dossiers that reduce the time and cost for market authorization.
If you are a biotech company dealing with inaccurate drug screening results — this project developed standard operating procedures for stem cell-derived neuronal models and 3D brain organoids that provide proven relevance for clinical symptoms.
If you are a CRO dealing with small patient pools in rare disease trials — this project developed designs for innovative basket clinical trials that recruit patients with different disorders based on overlapping molecular paths.
By focusing on drug repurposing rather than traditional development, the project reduces the time and costs associated with regulatory approval and market authorization.
The project provides standard operating procedures for high-throughput drug screens using stem cell-derived models, though it is currently at the proof-of-concept stage for simultaneous development.
The project is developing blueprints for intellectual property strategies and business models through co-creation with partners.
SIMPATHIC is creating specific blueprints for regulatory dossiers to streamline the path for repurposed drugs across multiple indications.
The project period runs from 2023-07-01 to 2028-06-30.
The consortium is highly diversified with 21 partners across 10 countries. It maintains a healthy industrial presence with 4 industry partners (19% ratio), including 4 SMEs, balancing academic research from 8 universities and 4 research institutes with practical commercial application.
Contact Stichting Radboud Universitair Medisch Centrum in the Netherlands
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